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Archive for April, 2013

Drug Eluting Stents: On MIT’s Edelman Lab’s Contributions to Vascular Biology and its Pioneering Research on DES

Drug Eluting Stents: On MIT‘s Edelman Lab’s Contributions to Vascular Biology and its Pioneering Research on DES

Author: Larry H Bernstein, MD, FACP

and 

Curator: Aviva Lev-Ari, PhD, RN
http://PharmaceuticalIntelligence.com/2013/04/25/Contributions
-to-vascular-biology/

This is the first of a three part series on the evolution of vascular biology and the studies of the effects of biomaterials in vascular reconstruction and on drug delivery, which has embraced a collaboration of cardiologists at Harvard Medical School , Affiliated Hospitals, and MIT,
requiring cardiovascular scientists at the PhD and MD level, physicists, and computational biologists working in concert, and
an exploration of the depth of the contributions by a distinguished physician, scientist, and thinker.

The first part – Vascular Biology and Disease – will cover the advances in the research on

  • vascular biology,
  • signaling pathways,
  • drug diffusion across the endothelium and
  • the interactions with the underlying muscularis (media),
  • with additional considerations for type 2 diabetes mellitus.

The second part – Stents and Drug Delivery – will cover the

  • purposes,
  • properties and
  • evolution of stent technology with
  • the acquired knowledge of the pharmacodynamics of drug interactions and drug distribution.

The third part – Problems and Promise of Biomaterials Technology – will cover the shortcomings of the cardiovascular devices, and opportunities for improvement

Vascular Biology and Cardiovascular Disease

Early work on endothelial injury and drug release principles

The insertion of a catheter for the administration of heparin is not an innocuous procedure. Heparin is infused to block coagulation, lowering the risk of a dangerous

  • clot formation and
  • dissemination.

It was shown experimentally that the continuous infusion of heparin

  • suppresses smooth muscle proliferation after endothelial injury. It may lead to
  • hemorrhage as a primary effect.

The anticoagulant property of heparin was removed by chemical modification without loss of the anti-proliferative effect.

In this study, MIT researches placed ethylene-vinyl acetate copolymer matrices containing standard and modified heparin adjacent to rat carotid arteries at the time of balloon deendothelialization.

Matrix delivery of both heparin compounds effectively diminished this proliferation in comparison to controls without producing systemic anticoagulation or side effects.

This mode of therapy appeared more effective than administering the agents by either

  • intravenous pumps or
  • heparin/polymer matrices placed in a subcutaneous site distant from the injured carotid artery

This indicated that the site of placement at the site of injury is a factor in the microenvironment, and is a preference for avoiding restenosis after angioplasty and other interventions.

This raised the question of why the proliferation of vascular muscle occurs in the first place.
 Edelman, Nugent and Karnovsky  (1) showed that the proliferation required first the denudation of vascular surface endothelium. This exposed the underlayer to the effect of basic fibroblast growth factor, which stimulates mitogenesis of the exposed cell, explained by the endothelium as a barrier from circulating bFGF.

To answer this question, they compared the effect of

  • 125I-labelled bFGF intravenously given with perivascular controlled bFGF release.
  • Polymeric controlled release devices delivered bFGF to the extravascular space without transendothelial transport. 
Deposition within the blood vessel wall was rapidly distributed circumferentially and was substantially greater than that observed following intravenous injection.

The amount of bFGF deposited in arteries adjacent to the release devices was 40 times that deposited in similar arteries in animals who received a single intravenous bolus of bFGF.

The presence of intimal hyperplasia increased deposition of perivascularly released bFGF 2.4-fold but decreased the deposition of intravenously injected bFGF by 67%.

  • bFGF was 5- to 30-fold more abundant in solid organs after intravenous injection than it was following perivascular release, and
  • bFGF deposition was greatest in the kidney, liver, and spleen and was substantially lower in the heart and lung.

This result indicated that vascular deposition of bFGF is independent of endothelium, and

  • bFGF delivery is effectively perivascular. (2)

Drug activity studies have to be done in well controlled and representative conditions.
 Edelsman’s Lab researchers studied the

  • dose response of injured arteries to exogenous heparin in vivo by providing steady and predictable arterial levels of drug.
  • Controlled-release devices were fabricated to direct heparin uniformly and at a steady rate to the adventitial surface of balloon-injured rat carotid arteries.

Researchers predicted the distribution of heparin throughout the arterial wall using computational simulations and correlated these concentrations with the biologic response of the tissues.

Researchers determined from this process that an in vivo arterial concentration of 0.3 mg/ml of heparin is required to maximallyinhibit intimal hyperplasia after injury.

This estimation of the required tissue concentration of a drug is

  • independent of the route of administration and
  • applies to all forms of drug release.

In this way the Team was able to

  • evaluate the potential of  widely disparate forms of drug release and, to finally
  • create some rigorous criteria by which to guide the development of particular delivery strategies for local diseases. (3)

Chiefly, the following three effects:

(1) Effect of controlled adventitial heparin delivery on smooth muscle cell proliferation following endothelial injury. ER Edelman, DH Adams, and MJ Karnovsky. PNAS May 1990; 87: 3773-3777.


(2) Perivascular and intravenous administration of basic fibroblast growth factor: Vascular and solid organ deposition. ER Edelman, MA Nugent, and MJ Karnovsky. PNAS Feb 1993; 90: 1513-1517.


(3) Tissue concentration of heparin, not administered dose, correlates with the biological response of injured arteries in vivo. MA Lovich and ER Edelman. PNAS Sep 1999; 96: 11111–11116.

Vascular Injury and Repair

Perlecan is a heparin-sulfate proteoglycan that might be critical for regulation of vascular repair by inhibiting the binding and mitogenic activity of basic fibroblast growth factor-2 (bFGF-2) in vascular smooth muscle cells .

The Team generated

  • Clones of endothelial cells expressing an antisense vector targeting domain III of perlecan. The transfected cells produced significantly less perlecan than parent cells, and they had reduced bFGF in vascular smooth muscle cells.
  • Endothelial cells were seeded onto three-dimensional polymeric matrices and implanted adjacent to porcine carotid arteries subjected to deep injury.
  • The parent endothelial cells prevented thrombosis, but perlecan deficient cells were ineffective.

The ability of endothelial cells to inhibit intimal hyperplasia, however, was only in part suppressed by perlecan. The differential regulation by perlecan of these aspects of vascular repair may clarify why control of clinical clot formation does not lead to full control of intimal hyperplasia.

The use of genetically modified tissue engineered cells provides a new approach for dissecting the role of specific factors within the blood vessel wall.(1) Successful implementation of local arterial drug delivery requires transmural distribution of drug. The physicochemical properties of the applied compound govern its transport and tissue binding.

  • Hydrophilic compounds are cleared rapidly.
  • Hydrophobic drugs bind to fixed tissue elements, potentially prolonging tissue residence and biological effect.

Local vascular drug delivery provides

  • elevated concentrations of drug in the target tissue while
  • minimizing systemic side effects.

To better characterize local pharmacokinetics the Team examined the arterial transport of locally applied dextran and dextran derivatives in vivo.

Using a two-compartment pharmacokinetic model to correct

  • The measured transmural flux of these compounds for systemic
  • Redistribution and elimination as delivered from a photo-polymerizable hydrogel.
  • The diffusivities and the transendothelial permeabilities were strongly dependent on molecular weight and charge
  • For neutral dextrans, the diffusive resistance increased with molecular weightapproximately 4.1-fold between the molecular weights of 10 and 282 kDa.
  • Endothelial resistance increased 28-fold over the same molecular weight range.
  • The effective medial diffusive resistance was unaffected by cationic charge as such molecules moved identically to neutral compounds, but increased approximately 40% when dextrans were negatively charged.

Transendothelial resistance was 20-fold lower for the cationic dextrans, and 11-fold higher for the anionic dextrans, when both were compared to neutral counterparts.

These results suggest that, while

  • low molecular weight drugs will rapidly traverse the arterial wall with the endothelium posing a minimal barrier,
  • the reverse is true for high molecular weight agents.

The deposition and distribution of locally released vascular therapeutic compounds might be predicted based upon chemical properties, such as molecular weight and charge. (2)

Paclitaxel is hydrophobic and has therapeutic potential against proliferative vascular disease.
 The favorable preclinical data with this compound may, in part, result from preferential tissue binding.
 The complexity of Paclitaxel pharmacokinetics required in-depth investigation if this drug is to reach its full clinical potential in proliferative vascular diseases.

Equilibrium distribution of Paclitaxel reveals partitioning above and beyond perfusate concentration and a spatial gradient of drug across the arterial wall.

The effective diffusivity (Deff) was estimated from the Paclitaxel distribution data to

  • facilitate comparison of transport of Paclitaxel through arterial parenchyma with that of other vasoactive agents and to
  • characterize the disparity between endovascular and perivascular application of drug.

This transport parameter described the motion of drug in tissues given an applied concentration gradient and includes, in addition to diffusion,

  • the impact of steric hindrance within the arterial interstitium;
  • nonspecific binding to arterial elements; and, in the preparation used here,
  • convective effects from the applied transmural pressure gradient.

At all times, the effective diffusivity for endovascular delivery exceeded that of perivascular delivery. The arterial transport of Paclitaxel was quantified through application ex vivo and measurement of the subsequent transmural distribution.

  • Arterial Paclitaxel deposition at equilibrium varied across the arterial wall.
  • Permeation into the wall increased with time, from 15 minutes to 4 hours, and
  • varied with the origin of delivery.

In contrast to hydrophilic compounds, the concentration in tissue exceeded the applied concentration and the rate of transport was markedly slower. Furthermore, endovascular and perivascular Paclitaxel application led to differences in deposition across the blood vessel wall.

This leads to a conclusion that Paclitaxel interacts with arterial tissue elements  as it moves under the forces of

  • diffusion and
  • convection and
  • can establish substantial partitioning and spatial gradients across the tissue. (3)

Endovascular drug-eluting stents have changed the practice of  cardiovascular vascularization, and yet it is unclear how they so dramatically reduce restenosis

We don’t know how to distinguish between the different formulations available.
 Researchers are now questioning whether individual properties of different drugs beyond lipid avidity effect arterial transport and distribution.

In bovine internal carotid segments, tissue-loading profiles for

  • Hydrophobic Paclitaxel and Rapamycin are indistinguishable, reaching load steady state after 2 days.
  • Hydrophilic dextran reaches equilibrium in hours.

Paclitaxel and Rapamycin bind to the artery at 30–40 times bulk concentration, and bind to specific tissue elements.

Transmural drug distribution profiles are markedly different for the two compounds.

  • Rapamycin binds specifically to FKBP12 binding protein and it distributes evenly through the artery,
  • Paclitaxel binds specifically to microtubules, and remains primarily in the subintimal space.

The binding of Rapamycin and Paclitaxel to specific intracellular proteins plays an essential role in

  • determining arterial transport and distribution and in
  • distinguishing one compound from another.

These results offer further insight into the

  • mechanism of local drug delivery and the
  • specific use of existing drug-eluting stent formulations. (4)

The Role of Amyloid beta (A) in Creation of Vascular Toxic Plaque

Amyloid beta (A) is a peptide family produced and deposited in neurons and endothelial cells (EC).
It is found at subnanomolar concentrations in the plasma of healthy individuals.
 Simple conformational changes produce a form of A-beta , A-beta 42, which creates toxic plaque in the brains of Alzheimer’s patients.

Oxidative stress induced blood brain barrier degeneration has been proposed as a key factor for A-beta 42 toxicity.

This cannot account for lack of injury from the same peptide in healthy tissues.
Researchers hypothesized that cell state mediates A-beta’s effect.
 They examined the viability in the presence of A-beta secreted from transfected
Chinese hamster ovary cells (CHO) of

  • aortic Endothelial Cells (EC),
  • vascular smooth muscle cells (SMC) and
  • epithelial cells (EPI) in different states

A-beta was more toxic to all cell types when they were subconfluent.
 Subconfluent EC sprouted and SMC and EPI were inhibited by A-beta.
Confluent EC were virtually resistant to A-beta and suppressed A-beta production by A-beta +CHO.

Products of subconfluent EC overcame this resistant state, stimulating the production and toxicity of A-beta 42. Confluent EC overgrew >35% beyond their quiescent state in the presence of A-beta conditioned in media from subconfluent EC.

These findings imply that A-beta 42 may well be even more cytotoxic to cells in injured or growth states and potentially explain the variable and potent effects of this protein.

One may now need to consider tissue and cell state in addition to local concentration of and exposure duration to A-beta.

The specific interactions of A-beta and EC in a state-dependent fashion may help understand further the common and divergent forms of vascular and cerebral toxicity of A-beta and the spectrum of AD. (5)

(1) Perlecan is required to inhibit thrombosis after deep vascular injury and contributes
to endothelial cell-mediated inhibition of intimal hyperplasia. MA Nugent, HM Nugent,
RV Iozzoi, K Sanchack, and ER Edelman. PNAS Jun 2000; 97(12): 6722-6727


(2) Correlation of transarterial transport of various dextrans with their physicochemical properties.
O Elmalak, MA Lovich, E Edelman. Biomaterials 2000; 21: 2263-2272


(3) Arterial Paclitaxel Distribution and Deposition. CJ Creel, MA Lovich, ER Edelman. Circ Res. 2000;86:879-884


(4) Specific binding to intracellular proteins determines arterial transport properties for rapamycin and Paclitaxel.
AD Levin, N Vukmirovic, Chao-Wei Hwang, and ER Edelman. PNAS Jun 2004; 101(25): 9463–9467.
www.pnas.org/cgi/doi/10.1073/pnas.0400918101

(5) Amyloid beta toxicity dependent upon endothelial cell state. M Balcells, JS Wallins, ER Edelman.
Neuroscience Letters 441 (2008) 319–322

Endothelial Damage as an Inflammatory State

Autoimmunity may drive vascular disease through anti-endothelial cell (EC) antibodies. This raises a question about whether an increased morbidity of cardiovascular diseases in concert with systemic illnesses may involve these antibodies.

Matrix-embedded ECs act as powerful regulators of vascular repair accompanied by significant reduction in expected systemic and local inflammation.

The Lab researchers compared the immune response against free and matrix-embedded ECs in naive mice and mice with heightened EC immune reactivity. Mice were presensitized to EC with repeated subcutaneous injections of saline-suspended porcine EC (PAE) (5*10^5 cells).

On day 42, both naive mice (controls) and mice with heightened EC immune reactivity received 5*10^5 matrix-embedded or free PAEs. Circulating PAE-specific antibodies and effector T-cells were analyzed 90 days after implantation for –

  • PAE-specific antibody-titers,
  • frequency of CD4+-effector cells, and
  • xenoreactive splenocytes

These were 2- to 4-fold lower (P<0.0001) when naıve mice were injected with matrix-embedded instead of saline-suspended PAEs.

Though basal levels of circulating antibodies were significantly elevated after serial PAE injections (2210+341 mean fluorescence intensity, day 42) and almost doubled again 90 days after injection of a fourth set of free PAEs, antibody levels declined by half in recipients of matrix-embedded PAEs at day 42 (P<0.0001), as did levels of CD4+-effector cells and xenoreactive splenocytes.

A significant immune response to implantation of free PAE is elicited in naıve mice, that is even more pronounced in mice with pre-developed anti-endothelial immunity.

Matrix-embedding protects xenogeneic ECs against immune reaction in naive mice and in mice with heightened immune reactivity.

Matrix-embedded EC might offer a promising approach for treatment of advanced cardiovascular disease. (1)

Researchers examined the molecular mechanisms through which

mechanical force and hypertension modulate

endothelial cell regulation of vascular homeostasis.

Exposure to mechanical strain increased the paracrine inhibition of vascular smooth muscle cells (VSMCs) by endothelial cells.

Mechanical strain stimulated the production by endothelial cells of perlecan and heparan-sulfate glycosaminoglycans. By inhibiting the expression of perlecan with an antisense vector researchers demonstrated that perlecan was essential to the strain-mediated effects on endothelial cell growth control.

Mechanical regulation of perlecan expression in endothelial cells was

  • governed by a mechano-transduction pathway
  • requiring transforming growth factor (TGF-β) signaling and
  • intracellular signaling through the ERK pathway.

Immunohistochemical staining of the aortae of spontaneously hypertensive rats
demonstrated strong correlations between

  • endothelial TGF-β,
  • phosphorylated signaling intermediates, and
  • arterial thickening.

Studies on ex vivo arteries exposed to varying levels of pressure demonstrated that

ERK and TGF-beta signaling were required for pressure-induced upregulation of endothelial HSPG.

The Team’s findings suggest a novel feedback control mechanism in which

  • net arterial remodeling to hemodynamic forces is controlled by a dynamic interplay between growth stimulatory signals from vSMCs and
  • growth inhibitory signals from endothelial cells. (2)

Heparan-sulfate proteoglycans (HSPGs) are potent regulators of vascular remodeling and repair.
 The major enzyme capable of degrading HSPGs is heparanase, which led us to examine
the role of heparanase in controlling

  • arterial structure,
  • mechanics, and
  • remodeling.

In vitro studies suggested heparanase expression in endothelial cells serves as a negative regulator of endothelial inhibition of vascular smooth muscle cell (vSMC) proliferation.

ECs inhibit vSMC proliferation through the interplay between

  • growth stimulatory signals from vSMCs and
  • growth inhibitory signals from ECs.

This would be expected if ECs had HSPGs that are degraded by heparanase.
Arterial structure and remodeling to injury is modified by heparanase expression.
Transgenic mice overexpressing heparanase had

  • increased arterial thickness,
  • cellular density, and
  • mechanical compliance.

Endovascular stenting studies in Zucker rats demonstrated increased heparanase expression in the neointima of obese, hyperlipidemic rats in comparison to lean rats.

The extent of heparanase expression within the neointima strongly correlated with the neointimal thickness following injury. To test the effects of heparanase overexpression on arterial repair, researchers developed a novel murine model of stent injury using small diameter self-expanding stents.

Using this model, researchers found that increased

  • neointimal formation and
  • macrophage recruitment occurs in transgenic mice overexpressing heparanase.
  • Taken together, these results support a role for heparanase in the regulation of arterial structure, mechanics, and repair. (3)

The first host–donor reaction in transplantation occurs at the blood–tissue interface.
When the primary component of the implant (donor) is the endothelial cells, it incites an immunologic reaction. Injections of free endothelial cell implants elicit a profound major histocompatibility complex (MHC) II dominated immune response.

Endothelial cells embedded within three-dimensional matrices behave like quiescent endothelial cells.

Perivascular implants of such embedded ECs cells are the most potent inhibitor of intimal hyperplasia and thrombosis following controlled vascular injury, but without any immune reactivity.

Allo- and even exenogenic endothelial cells evoke no significant humoral or
cellular immune response in immune-competent hosts when embedded within matrices.
 Moreover,  endothelial implants are immune-modulatory, reducing the extent of the memory response to previous free cell implants.

Attenuated immunogenicity results in muted activation of adaptive and innate immune cells. These findings point toward a pivotal role of matrix–cell-interconnectivity for

  • the cellular immune phenotype and might therefore assist in the design  of
  • extracellular matrix components for successful tissue engineering. (4)

Because changes in subendothelial matrix composition are associated with alterations of the endothelial immune phenotype, researchers sought to understand if

  • cytokine-induced NF-κB activity and
  • downstream effects depend on substrate adherence of endothelial cells (EC).

The team compared the upstream

  • phosphorylation cascade,
  • activation of NF-ĸβ, and
  • expression/secretion

of downstream effects of EC grown on tissue culture polystyrene plates (TCPS) with EC embedded within collagen-based matrices (MEEC).

Adhesion of natural killer (NK) cells was quantified in vitro and in vivo.

  • NF-κβ subunit p65 nuclear levels were significantly lower and
  • p50 significantly higher in cytokine-stimulated MEEC than in EC-TCPS.

Despite similar surface expression of TNF-α receptors, MEEC had significantly decreased secretion and expression of IL-6, IL-8, MCP-1, VCAM-1, and ICAM-1.

Attenuated fractalkine expression and secretion in MEEC (two to threefold lower than in EC-TCPS; p < 0.0002) correlated with 3.7-fold lower NK cell adhesion to EC (6,335 ± 420 vs. 1,735 ± 135 cpm; p < 0.0002).

Furthermore, NK cell infiltration into sites of EC implantation in vivo was significantly reduced when EC were embedded within matrix.

Matrix embedding enables control of EC substratum interaction.

This in turn regulates chemokine and surface molecule expression and secretion, in particular – of those compounds within NF-κβ pathways,

  • chemoattraction of NK cells,
  • local inflammation, and
  • tissue repair. (5)

Monocyte recruitment and interaction with the endothelium is imperative to vascular recovery.

Tie2 plays a key role in endothelial health and vascular remodeling.
Researchers studied monocyte-mediated Tie2/angiopoietin signaling following interaction of primary monocytes with endothelial cells and its role in endothelial cell survival.

The direct interaction of primary monocytes with subconfluent endothelial cells

resulted in transient secretion of angiopoietin-1 from monocytes and

the activation of endothelial Tie2. This effect was abolished by preactivation of monocytes with tumor necrosis factor-α (TNFα).

Although primary monocytes contained high levels of

  • both angiopoietin 1 and 2,
  • endothelial cells contained primarily angiopoietin 2.

Seeding of monocytes on serum-starved endothelial cells reduced caspase-3 activity by 46+5.1%, and 52+5.8% after TNFα treatment, and it decreased single-stranded DNA levels by 41+4.2% and 40+ 3.5%, respectively.

This protective effect of monocytes on endothelial cells was reversed by Tie2 silencing with specific short interfering RNA.

The antiapoptotic effect of monocytes was further supported by the

  • activation of cell survival signaling pathways involving phosphatidylinositol 3-kinase,
  • STAT3, and
  • AKT.

Monocytes and endothelial cells form a unique Tie2/angiopoietin-1 signaling system that affects endothelial cell survival and may play critical a role in vascular remodeling and homeostasis. (6)

(1) Cell–Matrix Contact Prevents Recognition and Damage of Endothelial Cells in States of Heightened Immunity.
H Methe, ER Edelman. Circulation. 2006;114[suppl I]:I-233–I-238.
http://www.circulationaha.org/DOI/10.1161/CIRCULATIONAHA.105.000687

(2) Endothelial Cells Provide Feedback Control for Vascular Remodeling Through a Mechanosensitive Autocrine
TGFβ Signaling Pathway. AB Baker, DS Ettenson, M Jonas, MA Nugent, RV Iozzo, ER Edelman.
Circ. Res. 2008;103;289-297   http://dx.doi.org/10.1161/CIRCRESAHA.108.179465http://circres.ahajournals.org/cgi/content/full/103/3/289

(3) Heparanase Alters Arterial Structure, Mechanics, and Repair Following Endovascular Stenting in Mice.
AB Baker, A Groothuis, M Jonas, DS Ettenson…ER Edelman.   Circ. Res. 2009;104;380-387;
http://dx.doi.org/10.1161/CIRCRESAHA.108.180695  http://circres.ahajournals.org/cgi/content/full/104/3/380

(4) The effect of three-dimensional matrix-embedding of endothelial cells on the humoral and cellular immune response.
H Methe, S Hess, ER Edelman. Seminars in Immunology 20 (2008) 117–122. http://dx.doi.org/10.1016/j.smim.2007.12.005

(5) NF-kB Activity in Endothelial Cells Is Modulated by Cell Substratum Inter-actions and Influences Chemokine-Mediated
Adhesion of Natural Killer Cells.  S Hess, H Methe, Jong-Oh Kim, ER Edelman.
Cell Transplantation 2009; 18: 261–273


(6) Primary Monocytes Regulate Endothelial Cell Survival Through Secretion of Angiopoietin-1 and Activation of Endothelial Tie2.
SY Schubert, A Benarroch, J Monter-Solans and ER Edelman. Arterioscler Thromb Vasc Biol 2011;31;870-875
http://dx.doi.org/10.1161/ATVBAHA.110.218255

Neointimal Formation, Shear Stress, and Remodelling with Reference to Diabetes

Innate immunity is of major importance in vascular repair. The present study evaluated whether

  • systemic and transient depletion of monocytes and macrophages with
  • liposome-encapsulated bisphosphonates inhibits experimental in-stent neointimal formation.

The Experiment

Rabbits fed on a hypercholesterolemic diet underwent bilateral iliac artery balloon denudation and stent deployment.

Liposomal alendronate (3 or 6 mg/kg) was given concurrently with stenting.

  • Monocyte counts were reduced by 90% 24 to 48 hours aftera single injection of liposomal alendronate, returning to basal levels at 6 days.

This treatment significantly reduced

  • intimal area at 28 days, from 3.88+0.93 to 2.08+0.58 and 2.16 +0.62 mm2.
  • Lumen area was increased from 2.87+0.44 to 3.57­+0.65 and 3.45+0.58 mm2, and
  • arterial stenosis was reduced from 58 11% to 37 8% and 38 7% in controls, in rabbits treated with 3 mg/kg, and with 6 mg/kg, respectively (mean+SD, n=8 rabbits/group, P< 0.01 for all 3 parameters).

No drug-related adverse effects were observed.
Reduction in neointimal formation was associated with

  • reduced arterial macrophage infiltration and proliferation at 6 days and with an
  • equal reduction in intimal macrophage and smooth muscle cell content at 28 days after injury.

Conversely, drug regimens ineffective in reducing monocyte levels did not inhibit neointimal formation.
Researchers have shown that a

  • single liposomal bisphosphonates injection concurrent with injury reduces in-stent neointimal formation and
  • arterial stenosis in hypercholesterolemic rabbits, accompanied by systemic transient depletion of monocytes and macrophages. (1)

Diabetes and insulin resistance are associated with increased disease risk and poor outcomes from cardiovascular interventions.

Even drug-eluting stents exhibit reduced efficacy in patients with diabetes.
Researchers reported the first study of vascular response to stent injury in insulin-resistant and diabetic animal models.

Endovascular stents were expanded in the aortae of

  • obese insulin-resistant and
  • type 2 diabetic Zucker rats,
  • in streptozotocin-induced type 1 diabetic Sprague-Dawley rats, and
  • in matched controls.

Insulin-resistant rats developed thicker neointima (0.46+0.08 versus 0.37+0.06 mm2, P 0.05), with  decreased lumen area (2.95+0.26 versus 3.29+0.15 mm2, P 0.03) 14 days after stenting compared with controls, but without increased vascular inflammation (tissue macrophages).

Insulin-resistant and diabetic rat vessels did exhibit markedly altered signaling pathway activation 1 and 2 weeks after stenting, with up to a 98% increase in p-ERK (anti-phospho ERK) and a 54% reduction in p-Akt (anti-phospho Akt) stained cells. Western blotting confirmed a profound effect of insulin resistance and diabetes on Akt and ERK signaling in stented segments. p-ERK/p-Akt ratio in stented segments uniquely correlated with neointimal response (R2 = 0.888, P< 0.04) , but not in lean controls.

Transfemoral aortic stenting in rats provides insight into vascular responses in insulin resistance and diabetes.

Shifts in ERK and Akt signaling related to insulin resistance may reflect altered tissue repair in diabetes accompanied by a

  • shift in metabolic : proliferative balance.

These findings may help explain the increased vascular morbidity in diabetes and suggest specific therapies for patients with insulin resistance and diabetes. (2)

Researchers investigated the role of Valsartan (V) alone or in combination with Simvastatin (S) on coronary atherosclerosis and vascular remodeling, and tested the hypothesis that V or V/S attenuate the pro-inflammatory effect of low endothelial shear stress (ESS).

Twenty-four diabetic, hyperlipidemic swine were allocated into Early (n = 12) and Late (n=12) groups.
Diabetic swine in each group were treated with Placebo (n=4), V (n = 4) and V/S (n = 4) and  followed for 8 weeks in the Early group and 30 weeks in the Late group.

Blood pressure, serum cholesterol and glucose were similar across the treatment subgroups.
ESS was calculated in plaque-free subsegments of interest (n = 109) in the Late group at week 23.
Coronary arteries of this group were harvested at week 30, and the subsegments of interest were identified, and analyzed histopathologically.

Intravascular geometrically correct 3-dimensional reconstruction of the coronary arteries of 12 swine was performed 23 weeks after initiation of diabetes mellitus and a hyperlipidemic diet. Local endothelial shear stress was calculated

  • in plaque-free subsegments of interest (n=142) with computational fluid dynamics, and
  • the coronary arteries (n=31) were harvested and the same subsegments were identified at 30 weeks.

V alone or with S

  • reduced the severity of inflammation in high-risk plaques.
Both regimens attenuated the severity of enzymatic degradation of the arterial wall, reducing the severity of expansive remodeling.
  • attenuated the pro-inflammatory effect of low ESS.
V alone or with S
  • exerts a beneficial effect of reducing and stabilizing high-risk plaque characteristics independent of a blood pressure- and lipid-lowering effect. (3)

This study tested the hypothesis that low endothelial shear stress  augments the

  • expression of matrix-degrading proteases, promoting the
  • formation of thin-capped atheromata.

Researchers assessed the messenger RNA and protein expression, and elastolytic activity of selected elastases and their endogenous inhibitors.

Subsegments with low endothelial shear stress at week 23 showed

  • reduced endothelial coverage,
  • enhanced lipid accumulation, and
  • intense infiltration of activated inflammatory cells at week 30.

These lesions showed increased expression of messenger RNAs encoding

  • matrix metalloproteinase-2, -9, and -12, and cathepsins K and S
  • relative to their endogenous inhibitors and
  • increased elastolytic activity.

Expression of these enzymes correlated positively with the severity of internal elastic lamina fragmentation.

Thin-capped atheromata in regions with

  • lower preceding endothelial shear stress had
  • reduced endothelial coverage,
  • intense lipid and inflammatory cell accumulation,
  • enhanced messenger RNA expression and
  • elastolytic activity of MMPs and cathepsins with
  • severe internal elastic lamina fragmentation.

Low endothelial shear stress induces endothelial discontinuity and

  • accumulation of activated inflammatory cells, thereby
  • augmenting the expression and activity of elastases in the intima and
  • shifting the balance with their inhibitors toward matrix breakdown.

Team’s results provide new insight into the mechanisms of regional formation of plaques with thin fibrous caps. (4)

Elevated CRP levels predict increased incidence of cardiovascular events and poor outcomes following interventions. There is the suggestion that CRP is also a mediator of vascular injury.

Transgenic mice carrying the human CRP gene (CRPtg) are predisposed to arterial thrombosis post-injury.

Researchers examined whether CRP similarly modulates the proliferative and hyperplastic phases of vascular repair in CRPtg when thrombosis is controlled with daily aspirin and heparin at the time of trans-femoral arterial wire-injury.

Complete thrombotic arterial occlusion at 28 days was comparable for wild-type and CRPtg mice (14 and 19%, respectively). Neointimal area at 28d was 2.5 fold lower in CRPtg (4190±3134 m2, n = 12) compared to wild-types (10,157±8890 m2, n = 11, p < 0.05).

Likewise, neointimal/media area ratio was 1.10±0.87 in wild-types and 0.45±0.24 in CRPtg (p < 0.05).

  • Seven days post-injury, cellular proliferation and apoptotic cell number in the intima were both less pronounced in CRPtg than wild-type.
  • No differences were seen in leukocyte infiltration or endothelial coverage.
CRPtg mice had significantly reduced p38 MAPK signaling pathway activation following injury.

The pro-thrombotic phenotype of CRPtg mice was suppressed by aspirin/heparin, revealing CRP’s influence on neointimal growth after trans-femoral arterial wire-injury.

  • Signaling pathway activation,
  • cellular proliferation, and
  • neointimal formation

were all reduced in CRPtg following vascular injury.
 Increasingly the Team was aware of CRP multipotent effects.
 Once considered only a risk factor, and recently a harmful agent, CRP is a far more complex regulator of vascular biology. (5)

(1) Liposomal Alendronate Inhibits Systemic Innate Immunity and Reduces In-Stent Neointimal
Hyperplasia in Rabbits. HD Danenberg, G Golomb, A Groothuis, J Gao…, ER Edelman.
Circulation. 2003;108:2798-2804


(2) Vascular Neointimal Formation and Signaling Pathway Activation in Response to Stent Injury
in Insulin-Resistant and Diabetic Animals. M Jonas, ER Edelman, A Groothuis, AB Baker, P Seifert, C Rogers.
Circ. Res. 2005;97;725-733.        http://dx.doi.org/10.1161/01.RES.0000183730.52908.C6
http://circres.ahajournals.org/cgi/content/full/97/7/725

(3) Attenuation of inflammation and expansive remodeling by Valsartan alone or in combination with
Simvastatin in high-risk coronary atherosclerotic plaques. YS Chatzizisis, M Jonas, R Beigel, AU Coskun…
ER Edelman, CL Feldman, PH Stone.  Atherosclerosis 203 (2009) 387–394


(4) Augmented Expression and Activity of Extracellular Matrix-Degrading Enzymes in Regions of Low
Endothelial Shear Stress Colocalize With Coronary Atheromata With Thin Fibrous Caps in Pigs.
YS Chatzizisis, AB Baker, GK Sukhova,…P Libby, CL Feldman, ER Edelman, PH Stone
Circulation 2011;123;621-630     http://dx.doi.org/10.1161/CIRCULATIONAHA.110.970038
http://circ.ahajournals.org/cgi/content/full/123/6/621


(5) Neointimal formation is reduced after arterial injury in human crp transgenic mice
HD Danenberg, E Grad, RV Swaminathan, Z Chenc,…ER Edelman
Atherosclerosis 201 (2008) 85–91

A Rattle Bag of Science and the Art of Translation

Science Translational Medicine – A rattle bag of science and the art of translation
E. R. Edelman, G. A. FitzGerald.
Sci.Transl. Med. 3, 104ed3 (2011). http://dx.doi.org/10.1126/scitranslmed.3002131

Elazer R. Edelman is the Thomas D. and Virginia W. Cabot Professor of Health Sciences and Technology at MIT,
Professor of Medicine at Harvard Medical School, a coronary care unit cardiologist at the Brigham and Women’s
Hospital, and Director of the Harvard-MIT Biomedical Engineering Center. E-mail: ere@mit.edu

Garret A. FitzGerald is the McNeil Professor in Translational Medicine and Therapeutics, Chair of the Department of
Pharmacology, and Director of the Institute for Translational Medicine & Therapeutics, University of Pennsylvania.
E-mail: garret@upenn.edu

In 2011, the American Association for the Advancement of Science (AAAS)  founded Science Translational Medicine (STM)
to disseminate interdisciplinary science integrating basic and clinical research that defines and fosters new therapeutics, devices, and diagnostics.

Conceived and nourished under the creative vision of Elias Zerhouni and Katrina Kelner, the journal has attracted widespread attention.
Now, as we assume the mantle of co-chief scientific advisors, we look back on the journal’s early accomplishments, restate our mission, and make clear the kinds of manuscripts we seek and accept for publication.

STM’s mission, as articulated by Elias and Katrina, was to

“promote human health by providing a forum for communication and cross-fertilization among basic, translational, and clinical research practitioners and trainees from all relevant established and emerging disciplines.”

This statement remains relevant and accurate today.
 With this mission on our masthead, STM now receives ~25 manuscripts (full-length research articles) per week and publishes ~10% of them. Roughly half of the submissions are deemed inappropriate for the journal and are returned without review within 8 to 10 days of receipt.

Of those papers that undergo full peer review,

decisions to reject are made within 48 days and

the mean time to acceptance (including the revision period) is 125 days.

There is now an average wait of only 24 days between acceptance and publication.

Defining TRANSLATIONAL Medicine

In accord with the journal’s broad readership, the ideal manuscript meets five criteria: It
(i) reports a discovery of translational relevance with high-impact potential;
(ii) has a conceptual focus with interdisciplinary appeal;
(iii) elucidates a biological mechanism;
(iv) is innovative and novel; and
(v) is presented in clear, broadly accessible language.
 STM seeks to publish research that describes

  • how innovative concepts drive the creative biomedical science
  • that ultimately improves the quality of people’s lives—

This is the broadest of our journal’s criteria but is the one that sets us apart as well.
Translational relevance does not require demonstration of benefit in humans but does require the evident potential to advance clinical medicine, thus impacting the direction of our culture and the welfare of our communities. Conceptual focus and mechanistic emphasis discriminate our papers from those that contain observational descriptions of technical findings for which value is restricted to a specific discipline.

However, innovation and novelty may apply to a fundamental scientific discovery or to the nature of its application and relevance to the translational process. Criteria enable the journal to consider versatile technological advances that apply new and creative thinking but may not necessarily offer fresh insights into biological mechanisms. Finally, while the subsequent additional efforts of the STM editorial staff are not to be discounted, the clarity of writing and coherence of argument presented within a submitted manuscript are likely to facilitate its progress through the challenge of peer review.

On Causes – Hippocrates, Aristotle, Robert Koch, and the Dread Pirate Roberts

Elazer R. Edelman
Circulation 2001;104:2509-2512

The idea of risk factors for vascular disease has evolved

  • from a dichotomous to continuous hazard analysis and
  • from the consideration of a few factors to
  • mechanistic investigation of many interrelated risks.

However, confusion still abounds regarding issues of association and causation. Originally, the simple presence of

  • tobacco abuse, hypertension, and/or hypercholesterolemia were tallied, and
  • the cumulative score was predictive of subsequent coronary artery disease.

Since then, dose responses have been defined for these and other factors and it has been suggested that almost 300 factors place patients at risk; these factors include elevations in plasma homocysteine.
 Recent studies shed interesting light on the mechanism of this potentially causal relationship, which was first noted in 1969.

Aside from putative effects on vessel wall dynamics, there is now direct evidence that homocysteine is atherogenic. Twenty-fold increases in plasma homocysteine achieved by dietary manipulation of apoE–/– mice increased aortic root lesion size 2-fold and produced a prolonged chronic inflammatory mural response accompanied by elevations in vascular cell adhesion molecule-1 (VCAM) and tumor necrosis factor-a (TNF-a).

In long term followup, homocysteine levels elevated by

  • dietary supplementation with methionine or homocysteine
  • promoted lesion size and plaque fibrosis in these
  • atherosclerosis-prone mice early in life, but without influencing ultimate plaque burden as the animals aged.

A number of mechanisms were proposed by which homocysteine achieved this effect, including

  • promotion of inflammation,
  • regulation of lipoprotein metabolism, and
  • modification of critical biochemical pathways and
  • metabolites including nitric oxide (NO).

See p 2569
In the present issue of Circulation,

Stühlinger et al 7 advance these mechanistic insights one critical step further by defining homocysteine’s effects at an enzymatic level.

The group led by Lentz published an association between levels of the

  • endogenous inhibitor of Nirtic Oxide synthase,
  • asymmetric dimethyl arginine (ADMA), and
  • homocysteine in cultured endothelial cells and in the serum of cynomolgus monkeys.

Such an association is interesting because the L-arginine–NO synthase pathway seems to be a critical component in the full range of endothelial cell biology and vascular dysfunction.

Stühlinger et al 7  now show that increased cultured endothelial cell elaboration of ADMA by homocysteine and its precursor L-methionine is associated with a dose-dependent impairment of the activity of endothelial dimethylarginine dimethylaminohydrolase (DDAH), the enzyme that degrades ADMA. Homocysteine directly inhibited DDAH activity in a cell-free system by targeting a critical sulfhydryl group on this enzyme.

Thus, one could envision that the balance of cardiovascular health and disease could well be determined by the ability of an intact Nirtic Oxide synthase system to overcome environmental, dietary, and even genetic factors.

In patients with altered enzymatic defense systems,

  • elevated homocysteine,
  • oxidized lipoproteins,
  • inflammation, and other
  • vasotoxins

may dominate even the most potent defense mechanisms.
These studies raise a number of issues.
Do we need to add to our list of established cardiovascular risk factors to accommodate new findings and associations?
Is there a final common pathway for all risk factors or perhaps even a unified factor theory into which all potential risks can be grouped?
And, as always, should we consider Nirtic Oxide at the core of this universality?
Finally, should we change our focus altogether and speak not of risk factors but of

  • genetic predisposition,
  • extent of biochemical aberration, and
  • degree of physical damage?

Some would view these remarkable success stories and the repeated association of hyperhomocyst(e)inemia with coronary, cerebral, and peripheral vascular disease and simply advocate for increased folic acid intake for all.

Indeed, this intervention of negligible cost and

  • insignificant side effect is already partially in place;
  • many foods are fortified with folate to prevent congenital neural tube defects.

This reader considers the seminal work by Vernon Young and Yves Ingenbleek on the relationship between

  • S8 and regions distant from lava flows in Asia and Indian subcontinents,
  • where they have determined hyperhomocysteinemia and the consequence associated with:
  • veganism (not voluntary)
  • impaired methyl donor reactions and transsulfuration pathways (not corrected by B12, folate)
  • loss of lean body mass due to the constant relationship of S:N (insufficient from plant sources)

What happens, when we fail to continue to pursue causality,

  • the linkage of biological significance or scientific plausibility with
  • epidemiologically or statistically significant association?

In medicine, risk becomes the likelihood that people without a disease will acquire the disease through contact with factors thought to increase disease risk.

All of these risk factors are then, by nature, imprecise and nonspecific.
 They are stochastic measures of what will happen to normal people who fall into particular measures of these parameters.

The daring may be willing to accept these risks, citing friend and foe who live well beyond or for far lesser times than anticipated by risk alone. Such concerns may well become moot if we can simultaneously identify patients at risk

  • by linking phenotype with genotype,
  • gene expression with protein elaboration, and
  • environmental exposures with the biochemical consequences and
  • direct anatomic aberrations they induce.

This kind of characterization may well replace a family history of arterial disease as a rough estimate of

  • genotype,
  • serum cholesterol as an indirect measure of the health of lipoprotein metabolism,
  • serum glucose as a crude determinant of the ravages of diabetes mellitus,
  • blood pressure measurement as a marker of long-standing endogenous exposure to altered flow, and
  • tobacco abuse as a maker of long-standing exposure to exogenous toxins.

Rather than identifying patients on the basis of their serum cholesterol, we will have a direct measure of their

  • LDL receptor number,
  • internalization rate,
  • macrophage content in the blood vessel wall,
  • metalloproteinase activity, etc.
  • insulin receptor metabolism,
  • oxidative state, and
  • glycated burden.
  • Serum glucose will similarly give way to these tests

Evaluating a new way to open clogged arteries: Computational model offers insight into mechanisms of drug-coated balloons.

A new study from MIT analyzes the potential usefulness of a new treatment that combines the benefits of angioplasty balloons and drug-releasing stents, but may pose fewer risks. With this new approach, a balloon is inflated in the artery for only a brief period, during which it releases a drug that prevents cells from accumulating and clogging the arteries over time.
While approved for limited use in Europe, these drug-coated balloons are still in development in the United States and have not received FDA approval. The MIT study, which models the behavior of the balloons, should help scientists optimize their performance and aid regulators in evaluating their effectiveness and safety.
“Until now, people who evaluate such technology could not distinguish hype from promise,” says Elazer Edelman, the Thomas D. and Virginia W. Cabot Professor of Health Sciences and Technology and senior author of the paper describing the study, which appeared online recently in the journal Circulation.
Lead author of the paper is Vijaya Kolachalama, a former MIT postdoc who is now a principal member of the technical staff at the Charles Stark Draper Laboratory.
Edelman’s lab is investigating a possible alternative to the current treatments: drug-coated balloons. “We’re trying to understand how and when this therapy could work and identify the conditions in which it may not,” Kolachalama says. “It has its merits; it has some disadvantages.”

Modeling drug release

The drug-coated balloons are delivered by a catheter and inflated at the narrowed artery for about 30 seconds, sometimes longer. During that time, the balloon coating, containing a drug such as Zotarolimus, is released from the balloon. The properties of the coating allow the drug to be absorbed in the body’s tissues. Once the drug is released, the balloon is removed.
In their new study, Kolachalama, Edelman and colleagues set out to rigorously characterize the properties of the drug-coated balloons. After performing experiments in tissue grown in the lab and in pigs, they developed a computer model that explains the dynamics of drug release and distribution. They found that factors such as the size of the balloon, the duration of delivery time, and the composition of the drug coating all influence how long the drug stays at the injury site and how effectively it clears the arteries.
One significant finding is that when the drug is released, some of it sticks to the lining of the blood vessels. Over time, that drug is slowly released back into the tissue, which explains why the drug’s effects last much longer than the initial 30-second release period.
“This is the first time we can explain the reasons why drug-coated balloons can work,” Kolachalama says. “The study also offers areas where people can consider thinking about optimizing drug transfer and delivery.”

http://circ.ahajournals.org/content/127/20/2047.short  
http://www.mit.edu/people/vbk/Circulation_2013.pdf 
http://www.sciencedaily.com/…13/05/130521121513.ht…    
Circulation, 2013; 127 (20): 2047 – 2055
http://dx.doi.org/10.1161/CIRCULATIONAHA.113.002051;

 

Conclusion

MIT’s Edelman’s Lab conducted the pioneering work in Vascular biology, animal models of drug eluting stents and was at the forefront of Empirical Molecular Cardiology in its studies in vascular physiology, biology and biomaterials for medical devices.

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Suppression of JAK2/STAT3 Signaling Reduces End-to-End Arterial Anastomosis Induced Cell Proliferation in Common Carotid Arteries of Rats (plosone.org)

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The Heart Revolution By Kilmer McCully, Martha McCully

HarperCollinsPublishers, 1969

http://books.google.com/books?id=iYLbuZFxEt8C&pg=PR20&dq=New+York+Times+homocysteine+and+Cholesterol&hl=en&sa=X&ei=_0F7UfDRA8zB4APozIHQAQ&ved=0CEMQ6AEwAg

 

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http://pharmaceuticalintelligence.com/2012/12/23/heart-renewal-by-pre-existing-cardiomyocytes-source-of-new-heart-cell-growth-discovered/

Cardiovascular Risk Inflammatory Marker: Risk Assessment for Coronary Heart Disease and Ischemic Stroke – Atherosclerosis.

Aviva Lev-Ari, PhD, RN 10/30/2012
http://pharmaceuticalintelligence.com/2012/10/30/cardiovascular-risk-inflammatory-marker-risk-assessment-for-coronary-heart-disease-and-ischemic-stroke-atherosclerosis/

To Stent or Not? A Critical Decision

Aviva Lev-Ari, PhD, RN 10/23/2012
http://pharmaceuticalintelligence.com/2012/10/23/to-stent-or-not-a-critical-decision/

New Definition of MI Unveiled, Fractional Flow Reserve (FFR)CT for Tagging Ischemia

Aviva Lev-Ari, PhD, RN 8/27/2012
http://pharmaceuticalintelligence.com/2012/08/27/new-definition-of-mi-unveiled-fractional-flow-reserve-ffrct-for-tagging-ischemia/

Ethical Considerations in Studying Drug Safety — The Institute of Medicine Report

Aviva Lev-Ari, PhD, RN 8/23/2012
http://pharmaceuticalintelligence.com/2012/08/23/ethical-considerations-in-studying-drug-safety-the-institute-of-medicine-report/

New Drug-Eluting Stent Works Well in STEMI

Aviva Lev-Ari, PhD, RN 8/22/2012
http://pharmaceuticalintelligence.com/2012/08/22/new-drug-eluting-stent-works-well-in-stemi/

Expected New Trends in Cardiology and Cardiovascular Medical Devices

Aviva Lev-Ari, PhD, RN 8/17/2012
http://pharmaceuticalintelligence.com/2012/08/17/expected-new-trends-in-cardiology-and-cardiovascular-medical-devices/

Coronary Artery Disease – Medical Devices Solutions: From First-In-Man Stent Implantation, via Medical Ethical Dilemmas to Drug Eluting Stents

Aviva Lev-Ari, PhD, RN 8/13/2012

http://pharmaceuticalintelligence.com/2012/08/13/coronary-artery-disease-medical-devices-solutions-from-first-in-man-stent-implantation-via-medical-ethical-dilemmas-to-drug-eluting-stents/

Percutaneous Endocardial Ablation of Scar-Related Ventricular Tachycardia

Aviva Lev-Ari, PhD, RN 7/18/2012

http://pharmaceuticalintelligence.com/2012/07/18/percutaneous-endocardial-ablation-of-scar-related-ventricular-tachycardia/

Competition in the Ecosystem of Medical Devices in Cardiac and Vascular Repair: Heart Valves, Stents, Catheterization Tools and Kits for Open Heart and Minimally Invasive Surgery (MIS)

Aviva Lev-Ari, PhD, RN 6/22/2012

http://pharmaceuticalintelligence.com/2012/06/22/competition-in-the-ecosystem-of-medical-devices-in-cardiac-and-vascular-repair-heart-valves-stents-catheterization-tools-and-kits-for-open-heart-and-minimally-invasive-surgery-mis/

Global Supplier Strategy for Market Penetration & Partnership Options (Niche Suppliers vs. National Leaders) in the Massachusetts Cardiology & Vascular Surgery Tools and Devices Market for Cardiac Operating Rooms and Angioplasty Suites

Aviva Lev-Ari, PhD, RN 6/22/2012

http://pharmaceuticalintelligence.com/2012/06/22/global-supplier-strategy-for-market-penetration-partnership-options-niche-suppliers-vs-national-leaders-in-the-massachusetts-cardiology-vascular-surgery-tools-and-devices-market-for-car/

Blood_Vessels

Blood_Vessels (Photo credit: shoebappa)

Visceral Myopathy in Statins

Visceral Myopathy in Statins (Photo credit: Snipergirl)

Medical science has advanced significantly sin...

Medical science has advanced significantly since 1507, when Leonardo da Vinci drew this diagram of the internal organs and vascular systems of a woman. (Photo credit: Wikipedia)

English: Lee Hood, MD, PhD, President and Co-f...

English: Lee Hood, MD, PhD, President and Co-found of the Institute for Systems Biology (Photo credit: Wikipedia)

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Economic Toll of Heart Failure in the US: Forecasting the Impact of Heart Failure in the United States – A Policy Statement From the American Heart Association

Reporter: Aviva Lev-Ari, PhD, RN

 

  • AHA Policy Statement

Forecasting the Impact of Heart Failure in the United States

A Policy Statement From the American Heart Association

  1. Paul A. Heidenreich, MD, MS, FAHA, Chair,

  2. Nancy M. Albert, PhD, RN, FAHA,
  3. Larry A. Allen, MD, MHS,
  4. David A. Bluemke, MD, PhD, FAHA,
  5. Javed Butler, MD, MPH, FAHA,
  6. Gregg C. Fonarow, MD, FAHA,
  7. John S. Ikonomidis, MD, PhD, FRCS(C), FAHA,
  8. Olga Khavjou, MA,
  9. Marvin A. Konstam, MD,
  10. Thomas M. Maddox, MD, MSc,
  11. Graham Nichol, MD, MPH, FRCP(C), FAHA,
  12. Michael Pham, MD, MPH,
  13. Ileana L. Piña, MD, MPH, FAHA,
  14. Justin G. Trogdon, PhD and
  15. on behalf of the American Heart Association Advocacy Coordinating Committee:
  • Council on Arteriosclerosis,
  • Thrombosis and Vascular Biology,
  • Council on Cardiovascular Radiology and Intervention,
  • Council on Clinical Cardiology,
  • Council on Epidemiology and Prevention, and
  • Stroke Council

Abstract

Background—Heart failure (HF) is an important contributor to both the burden and cost of national healthcare expenditures, with more older Americans hospitalized for HF than for any other medical condition. With the aging of the population, the impact of HF is expected to increase substantially.

Methods and Results—We estimated future costs of HF by adapting a methodology developed by the American Heart Association to project the epidemiology and future costs of HF from 2012 to 2030 without double counting the costs attributed to comorbid conditions. The model assumes that HF prevalence will remain constant by age, sex, and race/ethnicity and that rising costs and technological innovation will continue at the same rate.

By 2030,

  • >8 million people in the United States (1 in every 33) will have HF.
  • Between 2012 and 2030, real (2010$) total direct medical costs of HF are projected to increase from $21 billion to $53 billion.
  • Total costs, including indirect costs for HF, are estimated to increase from $31 billion in 2012 to $70 billion in 2030.
  • If one assumes all costs of cardiac care for HF patients are attributable to HF (no cost attribution to comorbid conditions), the 2030 projected cost estimates of treating patients with HF will be 3-fold higher ($160 billion in direct costs).

Conclusions—The estimated prevalence and cost of care for HF will increase markedly because of aging of the population. Strategies to prevent HF and improve the efficiency of care are needed.

Key Words:

http://circheartfailure.ahajournals.org/content/early/2013/04/24/HHF.0b013e318291329a.abstract

15 page PDF, at the below link

http://circheartfailure.ahajournals.org/content/early/2013/04/24/HHF.0b013e318291329a.full.pdf+html?sid=ad1efd74-a4e1-45b0-8a47-350e85435487

REFERENCE

Four Policy Statement From the American Heart Association

  1. AHA Policy StatementForecasting the Impact of Heart Failure in the United States: A Policy Statement From the American Heart Association

    • Paul A. Heidenreich,
    • Nancy M. Albert,
    • Larry A. Allen,
    • David A. Bluemke,
    • Javed Butler,
    • Gregg C. Fonarow,
    • John S. Ikonomidis,
    • Olga Khavjou,
    • Marvin A. Konstam,
    • Thomas M. Maddox,
    • Graham Nichol,
    • Michael Pham,
    • Ileana L. Piña,
    • and Justin G. Trogdon

    Circ Heart Fail. 2013;published online before print April 24 2013,doi:10.1161/HHF.0b013e318291329a

    …American Heart Association. Expert peer review of AHA Scientific Statements is conducted by the AHA Office of Science Operations…and improve the efficiency of care are needed. AHA Scientific Statements|heart failure|
  2. Select this article

    Special ReportStatement Regarding the Pre and Post Market Assessment of Durable, Implantable Ventricular Assist Devices in the United States

    • Michael A. Acker,
    • Francis D. Pagani,
    • Wendy Gattis Stough,
    • Douglas L. Mann,
    • Mariell Jessup,
    • Robert Kormos,
    • Mark S. Slaughter,
    • Timothy Baldwin,
    • Lynne Stevenson,
    • Keith D. Aaronson,
    • Leslie Miller,
    • David Naftel,
    • Clyde Yancy,
    • Joseph Rogers,
    • Jeffrey Teuteberg,
    • Randall C. Starling,
    • Bartley Griffith,
    • Steven Boyce,
    • Stephen Westaby,
    • Elizabeth Blume,
    • Peter Wearden,
    • Robert Higgins,
    • and Michael Mack

    Circ Heart Fail. 2013;6:e1-e11, published online before print November 12 2012,doi:10.1161/HHF.0b013e318279f6b5

    …wolterskluwer.com . Expert peer review of AHA Scientific Statements is conducted by the AHA Office of Science Operations…of Mechanically Assisted Circulatory Support.AHA Scientific Statements|heart-assist device|heart failure|BTC…
  3. Select this article

    Special ReportStatement Regarding the Pre and Post Market Assessment of Durable, Implantable Ventricular Assist Devices in the United States: Executive Summary

    • Michael A. Acker,
    • Francis D. Pagani,
    • Wendy Gattis Stough,
    • Douglas L. Mann,
    • Mariell Jessup,
    • Robert Kormos,
    • Mark S. Slaughter,
    • Timothy Baldwin,
    • Lynne Stevenson,
    • Keith D. Aaronson,
    • Leslie Miller,
    • David Naftel,
    • Clyde Yancy,
    • Joseph Rogers,
    • Jeffrey Teuteberg,
    • Randall C. Starling,
    • Bartley Griffith,
    • Steven Boyce,
    • Stephen Westaby,
    • Elizabeth Blume,
    • Peter Wearden,
    • Robert Higgins,
    • and Michael Mack

    Circ Heart Fail. 2013;6:145-150, published online before print November 12 2012,doi:10.1161/HHF.0b013e318279f55d

    …wolterskluwer.com . Expert peer review of AHA Scientific Statements is conducted by the AHA Office of Science Operations…of Mechanically Assisted Circulatory Support.AHA Scientific Statements|heart-assist device|heart failure| Background…
  4. Select this article

    ACCF/AHA/HFSA Data and Survey ReportACCF/AHA/HFSA 2011 Survey Results: Current Staffing Profile of Heart Failure Programs, Including Programs That Perform Heart Transplant and Mechanical Circulatory Support Device Implantation: A Report of the ACCF Heart Failure and Transplant Committee, AHA Heart Failure and Transplantation Committee, and Heart Failure Society of America

    • Mariell Jessup,
    • Nancy M. Albert,
    • David E. Lanfear,
    • JoAnn Lindenfeld,
    • Barry M. Massie,
    • Mary Norine Walsh,
    • and Mark J. Zucker

    Circ Heart Fail. 2011;4:378-387, published online before print April 4 2011,doi:10.1161/HHF.0b013e3182186210

    …hired for a given practice volume. These survey results are an initial step in developing such standards. AHA Scientific Statements|heart failure|heart transplant|mechanical circulatory support device|staffing profile| 1. Introduction…

http://circheartfailure.ahajournals.org/search?fulltext=AHA+Scientific+Statements&sortspec=date&submit=Submit&andorexactfulltext=phrase

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Reporter: Aviva Lev-Ari, PhD, RN

Centers of Excellence in Genomic Sciences (CEGS): NHGRI to Fund New Center (CEGS) on the Brain: Mental Disorders and the Nervous System

April 16, 2013

NEW YORK (GenomeWeb News) – The National Human Genome Research Institute plans to fund new Centers of Excellence in Genomic Sciences, or CEGS, to create interdisciplinary teams that pursue innovative genome-based approaches to address biomedical problems and to understanding the basis of biological systems.

NHGRI, along with support from the National Institute of Mental Health, expects to provide up to $2 million per year for each of the new CEGS it funds, and plans to award up to four new awards each year.

Although these CEGS may pursue a wide range of research objectives, NIMH will support the program because it wants to fund research using novel genomic approaches that can accelerate the understanding of the genetic basis of mental disorders and the nervous systemNHGRI said on Friday.

The CEGS program was created to use the new knowledge and technologies that resulted from the Human Genome Project and subsequent genomics research to develop new tools, methods, and concepts that apply to human biology and disease.
CEGS grantees are expected to be innovative, to focus on a critical issue in genomic science, to use multiple investigators working under one leader, to work toward a specific outcome, and to tackle challenging aspects of problems that may have impeded previous research efforts.

Further, they are supposed to bolster the pool of professional scientists and engineers who are trained in genomics through offering educational programs, and they are expected to address the shortage of scientists from underrepresented minority communities by developing recruiting programs that encourage minority community members to become independent genomics investigators.

The technologies and methods the CEGS investigators develop should be applicable to a wide range of cell types and organisms, and they should be scalable and expandable so they may apply to other model systems, according to NHGRI’s funding opportunity announcement.

Recent CEGS centers include

  • Caltech’s Center for In Toto Genomic Analysis of Vertebrate Development;
  • Harvard University’s Center for Transcriptional Consequences of Human Genetic Variation;
  • Johns Hopkins University’s Center for the Epigenetics of Common Human Disease;
  • Stanford University’s Center for the Genomic Basis of Vertebrate Diversity;
  • Arizona State University’s Microscale Life Sciences Center;
  • Medical College of Wisconsin, Milwaukee’s Center of Excellence in Genomics Science;
  • The University of North Carolina at Chapel Hill‘s CISGen center;
  • The Broad Institute’s Center for Cell Circuits;
  • Yale University’s Center for the Analysis of Human Genome Using Integrated Technologies; and
  • Dana-Farber Cancer Institute‘s Center for Genomic Analysis of Network Perturbations in Human Disease.

 http://www.genomeweb.com/nhgri-fund-new-centers-excellence-genomic-sciences

Center for In Toto Genomic Analysis of Vertebrate Development

P50 HG004071
Marianne Bronner-Fraser
California Institute of Technology, Pasadena, Calif.

This Center of Excellence in Genomic Science (CEGS) assembles a multidisciplinary group of investigators to develop innovative technologies with the goal of imaging and mutating every developmentally important vertebrate gene. Novel “in toto imaging” tools make it possible to use a systems-based approach for analysis of gene function in developing vertebrate embryos in real time and space. These tools can digitize in vivo data in a systematic, high-throughput, and quantitative fashion. Combining in toto imaging with novel gene traps permits a means to rapidly screen for developmentally relevant expression patterns, followed by the ability to immediately mutagenize genes of interest. Initially, key technologies will be developed and tested in the zebrafish embryo due to its transparency and the ability to obtain rapid feedback. Once validated, these techniques will be applied to an amniote, the avian embryo, due to several advantages including accessibility and similarity to human embryogenesis. Finally, to monitor alterations in gene expression in normal and mutant embryos, we will develop new techniques for in situ hybridization that permit simultaneous analysis of multiple marker genes in a sensitive and potentially quantitative manner. Our goal is to combine real time analysis of gene expression on a genome-wide scale coupled with the ability to mutate genes of interest and examine global alterations in gene expression as a result of gene loss. Much of the value will come from the development of new and broadly applicable technologies. In contrast to a typical technology development grant, however, there will be experimental fruit emerging from at least two vertebrate systems (zebrafish and avian). The following aims will be pursued: Specific Aim 1: Real-time “in toto” image analysis of reporter gene expression; Specific Aim 2: Comprehensive spatiotemporal analysis of gene function of the developing vertebrate embryo using the FlipTrap approach for gene trapping; Specific Aim 3: Design of quantitative, multiplexed ‘hybridization chain reaction’ (HCR) amplifiers for in vivo imaging with active background suppression; Specific Aim 4: Data analysis and integration of data sets to produce a “digital” fish and a “digital” bird. The technologies and the resulting atlases will be made broadly available via electronic publication.

Center Web Site: California Institute of Technology Center of Excellence in Genomic Science

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Causal Transcriptional Consequences of Human Genetic Variation

P50 HG005550
George M. Church
Harvard University, Cambridge, Mass.

The Center for Transcriptional Consequences of Human Genetic Variation (CTCHGV) will develop innovative and powerful genetic engineering methods and use them to identify genetic variations that causally control gene transcription levels. Genome Wide Association Studies (GWAS) find many variations associated with disease and other phenotypes, but the variations that may actually cause these conditions are hard to identify because nearby variations in the same haplotype blocks consistently co-occur with them in human populations, so that specifically causative ones cannot be distinguished. About 95% of GWAS variations are not in gene coding regions, and many of these presumably associate with altered gene expression levels. CTCHGV will identify the variations that directly control gene expression by engineering precise combinations of changes to gene regulatory regions that break down the haplotype blocks, allowing each variations’ effect on gene expression to be discerned independently of the others. To perform this analysis, CTCHGV will extract ~100kbps gene regulatory regions from human cell samples, create precise variations in them in E. coli, and re-introduce the altered regions back into human cells, using zinc finger nucleases (ZFNs) to efficiently induce recombination. CTCHGV will target 1000 genes for this analysis (Aim 1), and will use human induced Pluripotent Stem cells (iPS) to study the effects of variations in diverse human cell types (Aim 2). To explore the effects of variations in complex human tissues, CTCHGV will develop methods of measuring gene expression at transcriptome-wide levels in many single cells, including in situ in structured tissues (Aim 3). Finally, CTCHGV will develop novel advanced technologies that integrate DNA sequencing and synthesis to construct thousands of large DNA constructs from oligonucleotides, that enable very precise targeting and highly efficient performance of ZFNs, and that enable cells to be sorted on the basis of morphology as well as fluorescence and labeling (Aim 4). CTCHGV will also develop direct oligo-mediated engineering of human cells, and create “marked allele” iPS that will enable easy ascertainment of complete exon distributions for many pairs of gene alleles in many cell types.

Center Web Site: Center for Causal Transcriptional Consequences of Human Genetic Variation (CTCHGV)

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Center for the Epigenetics of Common Human Disease

P50 HG003233
Andrew P. Feinberg
Johns Hopkins University, Baltimore
(co-funded by National Institute of Mental Health)

Epigenetics, the study of non-DNA sequence-related heredity, is at the epicenter of modern medicine because it can help to explain the relationship between an individual’s genetic background, the environment, aging, and disease. The Center for the Epigenetics of Common Human Disease was created in 2004 to begin to develop the interface between epigenetics and epidemiologic-based phenotype studies, recognizing that epigenetics requires new ways of thinking about disease. We created a highly interdisciplinary group of faculty and trainees, including molecular biologists, biostatisticians, epidemiologists, and clinical investigators. We developed novel approaches to genome-wide DNA methylation (DNAm) analysis, allele-specific expression, and new statistical epigenetic tools. Using these tools, we discovered that most variable DNAm is in neither CpG islands nor promoters, but in what we term “CpG island shores,” regions of lower CpG density up to several kb from islands, and we have found altered DNAm in these regions in cancer, depression and autism. In the renewal period, we will develop the novel field of epigenetic epidemiology, the relationship between epigenetic variation, genetic variation, environment and phenotype. We will continue to pioneer genome-wide epigenetic technology that is cost effective for large scale analysis of population-based samples, applying our knowledge from the current period to second-generation sequencing for epigenetic measurement, including DNAm and allele-specific methylation. We will continue to pioneer new statistical approaches for quantitative and binary DNAm assessment in populations, including an Epigenetic Barcode. We will develop Foundational Epigenetic Epidemiology, examining: time-dependence, heritability and environmental relationship of epigenetic marks; heritability in MZ and DZ twins; and develop an epigenetic transmission disequilibrium test. We will then pioneer Etiologic Epigenetic Epidemiology, by integrating novel genome-wide methylation scans (GWMs) with existing Genome-Wide Association Study (GWAS) and epidemiologic phenotype data, a design we term Genome-Wide Integrated Susceptibility (GWIS), focusing on bipolar disorder, aging, and autism as paradigms for epigenetic studies of family-based samples, longitudinal analyses, and parent-of-origin effects, respectively. This work will be critical to realizing the full value of previous genetic and phenotypic studies, by developing and applying molecular and statistical tools necessary to integrate DNA sequence with epigenetic and environmental causes of disease.

Center Web Site: Center of Excellence in Genomic Science at Johns Hopkins

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Genomic Basis of Vertebrate Diversity

P50 HG002568
David M. Kingsley
Stanford University, Stanford, Calif.

The long-term goal of this project is to understand the genomic mechanisms that generate phenotypic diversity in vertebrates. Rapid progress in genomics has provided nearly complete sequences for several organisms. Comparative analysis suggests many fundamental pathways and gene networks are conserved between organisms. And yet, the morphology, physiology, and behavior of different species are obviously and profoundly different. What are the mechanisms that generate these key differences? Are unique traits controlled by few or many genetic changes? What kinds of changes? Are there particular genes and mechanisms that are used repeatedly when organisms adapt to new environments? Can better understanding of these mechanisms help explain dramatic differences in disease susceptibility that also exist between groups? The Stanford CEGS will use an innovative combination of approaches in fish, mice, and humans to identify the molecular basis of major phenotypic change in natural populations of vertebrates. Specific aims include: 1) cross stickleback fish and develop a genome wide map of the chromosomes, genes, and mutations that control a broad range of new morphological, physiological, and behavioral traits in natural environments; 2) test which population genetic measures provide the most reliable “signatures of selection” surrounding genes that are known to have served as the basis of parallel adaptive change in many different natural populations around the world; 3) assemble the stickleback proto Y chromosome and test whether either sex or autosomal rearrangements play an important role in generating phenotypic diversity, or are enriched in genomic regions that control phenotypic change; 4) test whether particular genes and mechanisms are used repeatedly to control phenotypic change in many different vertebrates. Preliminary data suggests that mechanisms identified as the basis of adaptive change in natural fish populations may be broadly predictive of adaptive mechanisms across a surprisingly large range of animals, including humans. Genetic regions hypothesized to be under selection in humans will be compared to genetic regions under selection in fish. Regions predicted to play an important role in natural human variation and disease susceptibility will be modeled in mice, generating new model systems for confirming functional variants predicted from human population genetics and comparative genomics.

Center Web Site: Stanford Genome Evolution Center

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Microscale Life Sciences Center

P50 HG002360
Deirdre R. Meldrum
Arizona State University, Tempe

Increasingly, it is becoming apparent that understanding, predicting, and diagnosing disease states is confounded by the inherent heterogeneity of in situ cell populations. This variation in cell fate can be dramatic, for instance, one cell living while an adjacent cell dies. Thus, in order to understand fundamental pathways involved in disease states, it is necessary to link preexisting cell state to cell fate in the disease process at the individual cell level.

The Microscale Life Sciences Center (MLSC) at the University of Washington is focused on solving this problem, by developing cutting-edge microscale technology for high throughput genomic-level and multi-parameter single-cell analysis, and applying that technology to fundamental problems of biology and health. Our vision is to address pathways to disease states directly at the individual cell level, at increasing levels of complexity that progressively move to an in vivo understanding of disease. We propose to apply MLSC technological innovations to questions that focus on the balance between cell proliferation and cell death. The top three killers in the United States, cancer, heart disease and stroke, all involve an imbalance in this cellular decision-making process. Because of intrinsic cellular heterogeneity in the live/die decision, this fundamental cellular biology problem is an example of one for which analysis of individual cells is essential for developing the link between genomics, cell function, and disease. The specific systems to be studied are proinflammatory cell death (pyroptosis) in a mouse macrophage model, and neoplastic progression in the Barrett’s Esophagus (BE) precancerous model. In each case, diagnostic signatures for specific cell states will be determined by measuring both physiological (cell cycle, ploidy, respiration rate, membrane potential) and genomic (gene expression profiles by single-cell proteomics, qRT-PCR and transcriptomics; LOH by LATE-PCR) parameters. These will then be correlated with cell fate via the same sets of measurements after a challenge is administered, for instance, a cell death stimulus for pyroptosis or a predisposing risk factor challenge (acid reflux) for BE. Ultimately, time series will be taken to map out the pathways that underlie the live/die decision.

Finally, this information will be used as a platform to define cell-cell interactions at the single-cell level, to move information on disease pathways towards greater in vivo relevance. New technology will be developed and integrated into the existing MLSC Living Cell Analysis cassette system to support these ambitious biological goals including 1) automated systems for cell placement, off-chip device interconnects, and high throughput data analysis with user friendly interfaces; 2) new optical and electronic sensors based on a new detection platform, new dyes and nanowires; and 3) new micromodules for single-cell qRT-PCR, LATE-PCR for LOH including single-cell pyrosequencing, on-chip single-cell proteomics, and single-cell transcriptomics using barcoded nanobeads.

Collaborating InstitutionsFred Hutchison Cancer Research Center, Brandeis University, University of Washington.

Center Web Site: Microscale Life Sciences Center

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Wisconsin Center of Excellence in Genomics Science

P50 HG004952
Michael Olivier
Medical College of Wisconsin, Milwaukee

The successful completion of the human genome and model organism sequences has ushered in a new era in biological research, with attention now focused on understanding the way in which genome sequence information is expressed and controlled. The focus of this proposed Wisconsin Center of Excellence in Genomics Science is to facilitate understanding of the complex and integrated regulatory mechanisms affecting gene transcription by developing novel technology for the comprehensive characterization and quantitative analysis of proteins interacting with DNA. This new technology will help provide for a genome-wide functional interpretation of the underlying mechanisms by which gene transcriptional regulation is altered during biological processes, development, disease, and in response to physiological, pharmacological, or environmental stressors. The development of chromatin immunoprecipitation approaches has allowed identification of the specific DNA sequences bound by proteins of interest. We propose to reverse this strategy and develop an entirely novel technology that will use oligonucleotide capture to pull down DNA sequences of interest, and mass spectrometry to identify and characterize the proteins and protein complexes bound and associated with particular DNA regions. This new approach will create an invaluable tool for deciphering the critical control processes regulating an essential biological function. The proposed interdisciplinary and multi-institutional Center of Excellence in Genomics Science combines specific expertise at the Medical College of Wisconsin, the University of Wisconsin Madison, and Marquette University. Technological developments in four specific areas will be pursued to develop this new approach: (1) cross-linking of proteins to DNA and fragmentation of chromatin; (2) capture of the protein-DNA complexes in a DNA sequence-specific manner; (3) mass spectrometry analysis to identify and quantify bound proteins; and (4) informatics to develop tools enabling the global analysis of the relationship between changes in protein-DNA interactions and gene expression. The Center will use carefully selected biological systems to develop and test the technology in an integrated genome-wide analysis platform that includes efficient data management and analysis tools. As part of the Center mission, we will combine our technology development efforts with an interdisciplinary training program for students and fellows designed to train qualified scientists experienced in cutting-edge genomics technology. Data, technology, and software will be widely disseminated by multiple mechanisms including licensing and commercialization activities.

Collaborating InstitutionsUniversity of Wisconsin-Madison, Marquette University

Center Web Site: Wisconsin Center of Excellence in Genomic Science

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CISGen

P50 MH090338
Fernando Pardo-Manuel de Villena
University of North Carolina, Chapel Hill

p>In this application, we propose a highly ambitious yet realistically attainable goal: to align existing expertise at UNC-Chapel Hill into a CEGS called CISGen. The overarching purpose of CISGen is to develop as a resource and to exploit the utility of the murine Collaborative Cross (CC) mouse model of the heterogeneous human population to delineate genetic and environmental determinants of complex phenotypes drawn from psychiatry, which are among the most intractable set of problems in all of biomedicine. Psychiatric disorders present a paradox – the associated morbidity, mortality, and costs are enormous and yet, despite over a century of scientific study, there are few hard facts about the etiology of the core diseases. Although our GWAS meta- analyses are in progress, early results suggest that strong and replicable findings may be elusive. Therefore, our proposal provides a complementary approach to the study of fundamental psychiatric phenotypes.

We propose a particularly challenging definition of success – we will identify high probability etiological models (which can be realistically complex) and then prove the predictive capacity of these models by generating novel strains of mice predicted to be at very high risk for the phenotype. Once validated, these high confidence models can then be tested in subsequent human studies – we do not propose human extension studies in CISGen but this is achievable for the investigators and their colleagues. Data collected in CISGen would be a valuable resource to the wider scientific community and could be applied to a large set of biological problems and these data can rapidly add to the knowledge base for any new genomewide association study (GWAS) finding. Delivery of sophisticated and user-friendly databases are a key component of CISGen.

Accomplishing this overarching goal requires an exceptional diversity of scientific expertise – psychiatry, human genetics, mouse behavior, mouse genetics, statistical genetics, computational biology, and systems biology. Experts in these disciplines are deeply involved in CISGen and are committed to the projects described herein. Successful integration of these diverse fields is non-trivial; however, all scientists on this application have had extensive interactions over the past five years, already know how to work together, and have a working knowledge of their colleagues’ expertise. UNC-Chapel Hill has an intense commitment to inter- disciplinary genomics research and provides a fertile backdrop for 21st century projects like CISGen.

Collaborating InstitutionsThe Jackson Laboratory, North Carolina State University, University of Texas at Arlington

Center Web Site: Center for Integrated Systems Genomics at UNC (CISGen)

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Center for Cell Circuits

P50 HG006193
Aviv Regev
The Broad Institute, Inc., Cambridge, Mass.

Systematic reconstruction of genetic and molecular circuits in mammalian cells remains a significant, largescale and unsolved challenge in genomics. The urgency to address it is underscored by the sizeable number of GWAS-derived disease genes whose functions remain largely obscure, limiting our progress towards biological understanding and therapeutic intervention. Recent advances in probing and manipulating cellular circuits on a genomic scale open the way for the development of a systematic method for circuit reconstruction. Here, we propose a Center for Cell Circuits to develop the reagents, technologies, algorithms, protocols and strategies needed to reconstruct molecular circuits. Our preliminary studies chart an initial path towards a universal strategy, which we will fully implement by developing a broad and integrated experimental and computational toolkit. We will develop methods for comprehensive profiling, genetic perturbations and mesoscale monitoring of diverse circuit layers (Aim 1). In parallel, we will develop a computational framework to analyze profiles, derive provisional models, use them to determine targets for perturbation and monitoring, and evaluate, refine and validate circuits based on those experiments (Aim 2). We will develop, test and refine this strategy in the context of two distinct and complementary mammalian circuits. First, we will produce an integrated, multi-layer circuit of the transcriptional response to pathogens in dendritic cells (Aim 3) as an example of an acute environmental response. Second, we will reconstruct the circuit of chromatin factors and non-coding RNAs that control chromatin organization and gene expression in mouse embryonic stem cells (Aim 4) as an example of the circuitry underlying stable cell states. These detailed datasets and models will reveal general principles of circuit organization, provide a resource for scientists in these two important fields, and allow computational biologists to test and develop algorithms. We will broadly disseminate our tools and methods to the community, enabling researchers to dissect any cell circuit of interest at unprecedented detail. Our work will open the way for reconstructing cellular circuits in human disease and individuals, to improve the accuracy of both diagnosis and treatment.

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Analysis of Human Genome Using Integrated Technologies

P50 HG002357
Michael P. Snyder
Yale University, New Haven, Conn.

We propose to establish a center to build genomic DNA arrays and develop novel technologies that will use these arrays for the large-scale functional analysis of the human genome. 0.3-1.4 kb fragments of nonrepetitive DNA from each of chromosomes 22, 21, 20, 19,7, 17, and perhaps the X chromosome will be prepared by PCR and attached to microscope slides. The arrays will be used to develop technologies for the large-scale mapping of 1) Transcribed sequences. 2) Binding sites of chromosomal proteins. 3) Origins of replication. 4) Genetic mutation and variation. A web-accessible database will be constructed to house the information generated in this study; data from other studies will also be integrated into the database. The arrays and technologies will be made available throughout both the Yale University and the larger scientific community. They will be integrated into our training programs for postdoctoral fellows, graduate students and undergraduates at Yale. We expect these procedures to be applicable to the analysis of the entire human genome and the genomes of many other organisms.

Center Web Site: Yale University Center for Excellence in Genomic Science

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Genomic Analysis of the Genotype-Phenotype Map

P50 HG002790
Simon Tavaré
University of Southern California, Los Angeles

Our Center, which started in 2003, focused on implications of haplotype structure in the human genome. Since that time, there have been extraordinary advances in genomics: Genome-wide association studies using single nucleotide polymorphisms and copy number variants are now commonplace, and we are rapidly moving towards whole-genome sequence data for large samples of individuals. Our Center has undergone similar dramatic changes. While the underlying theme remains the same — making sense of genetic variation — our focus is now explicitly on how we can use the heterogeneous data produced by modern genomics technologies to achieve such an understanding. The overall goal of our proposal is to develop an intellectual framework, together with computational and statistical analysis tools, for illuminating the path from genotype to phenotype, and for predicting the latter from the former. We will address three broad questions related to this problem: 1) How do we infer mechanisms by which genetic variation leads to changes in phenotype? 2) How do we improve the design, understanding and interpretation of association studies by exploiting prior information? 3) How do we identify general principles about the genotype-phenotype map? We will approach these questions through a series of interrelated projects that combine computational and experimental methods, explored in Arabidopsis, Drosophila and human, and involve a wide range of researchers including molecular biologists, population geneticists, genetic epidemiologists, statisticians, computer scientists, and mathematicians.

Collaborating InstitutionsUniversity of Utah

Center Web Site: The USC Center of Excellence in Genomic Science

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Genomic Analysis of Network Perturbations in Human Disease

P50 HG004233
Marc Vidal
Dana-Farber Cancer Institute, Boston

Genetic differences between individuals can greatly influence their susceptibility to disease. The information originating from the Human Genome Project (HGP), including the genome sequence and its annotation, together with projects such as the HapMap and the Human Cancer Genome Project (HCGP) have greatly accelerated our ability to find genetic variants and associate genes with a wide range of human diseases. Despite these advances, linking individual genes and their variations to disease remains a daunting challenge. Even where a causal variant has been identified, the biological insight that must precede a strategy for therapeutic intervention has generally been slow in coming. The primary reason for this is that the phenotypic effects of functional sequence variants are mediated by a dynamic network of gene products and metabolites, which exhibit emergent properties that cannot be understood one gene at a time. Our central hypothesis is that both human genetic variations and pathogens such as viruses influence local and global properties of networks to induce “disease states.” Therefore, we propose a general approach to understanding cellular networks based on environmental and genetic perturbations of network structure and readout of the effects using interactome mapping, proteomic analysis, and transcriptional profiling. We have chosen a defined model system with a variety of disease outcomes: viral infection. We will explore the concept that one must understand changes in complex cellular networks to fully understand the link between genotype, environment, and phenotype. We will integrate observations from network-level perturbations caused by particular viruses together with genome-wide human variation datasets for related human diseases with the goal of developing general principles for data integration and network prediction, instantiation of these in open-source software tools, and development of testable hypotheses that can be used to assess the value of our methods. Our plans to achieve these goals are summarized in the following specific aims: 1. Profile all viral-host protein-protein interactions for a group of viruses with related biological properties. 2. Profile the perturbations that viral proteins induce on the transcriptome of their host cells. 3. Combine the resulting interaction and perturbation data to derive cellular network-based models. 4. Use the developed models to interpret genome-wide genetic variations observed in human disease, 5. Integrate the bioinformatics resources developed by the various CCSG members within a Bioinformatics Core for data management and dissemination. 6. Building on existing education and outreach programs, we plan to develop a genomic and network centered educational program, with particular emphasis on providing access for underrepresented minorities to internships, workshop and scientific meetings.

Center Web SiteCenter for Cancer Systems Biology (CCSB) Center of Excellence in Genomic Science

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Reporter: Aviva Lev-Ari, PhD

 

April 25, 2013

Title: iRNA Agents with Biocleavable Tethers

Patent Number: 8,426,377

Filed: March 15, 2010

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

“The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety … [that] includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group,” the patent’s abstract states. “The cleavable linking group … is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent … to target cells, but … upon entry into a target cell is cleaved to release the iRNA agent from [a] targeting agent.”


Title: Inhibition of Viral Gene Expression Using Small Interfering RNA

Patent Number: 8,426,380

Filed: March 2, 2011

Lead Inventor: Roger Kaspar, Somagenics

“The invention provides methods, compositions, and kits comprising small interfering RNA that are useful for inhibition of viral-mediated gene expression,” the patent’s abstract states. “Small interfering RNAs as described herein can be used in methods of treatment of [hepatitis C virus] infection … [and include] shRNA and siRNA constructs targeting the internal ribosome entry site sequence of HCV.”


Title: In Vivo Polynucleotide Delivery Conjugates Having Enzyme-sensitive Linkages

Patent Number: 8,426,554

Filed: Dec. 23, 2011

Lead Inventor: David Rozema, Arrowhead Research

The invention, the patent’s abstract states, is “directed to compositions for delivery of RNA interference polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active polyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines are further covalently linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.”


Title: siRNA Targeting Myeloid Differentiation Primary Response Gene

Patent Number: 8,426,579

Filed: Aug. 11, 2011

Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)

“Efficient sequence-specific gene silencing is possible through the use of siRNA technology,” the patent’s abstract states. “By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for MYD88.”


Title: RNA Interference-mediated Inhibition of FC-Epsilon-R1-Alpha Gene

Patent Number: 8,426,581

Filed: Sept.12, 2011

Lead Inventor: Vasant Jadhav, Merck

The invention, the patent’s abstract states, relates to “compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases, and conditions that respond to the modulation of FC-epsilon-R1-alpha gene expression and/or activity, and/or modulate an FC-epsilon-R1-alpha gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules … that are capable of mediating or that mediate RNA interference against FC-epsilon-R1-alpha gene expression.”


Title: Methods for Producing microRNAs

Patent Number: 8,426,675

Filed: June 7, 2011

Lead Inventor: Ross Dickins, Cold Spring Harbor Laboratory

“The invention relates to recombinant vectors for inducible and/or tissue specific expression of double-stranded RNA molecules that interfere with the expression of a target gene,” according to the patent’s abstract. “In certain embodiments, the invention relates to the use of … tetracycline-responsive RNA polymerase II promoters to direct inducible knockdown in certain cells of an integrated or an endogenous gene, such as p53. The invention also relates to a method for producing transgenic animals expressing inducible, reversible, and/or tissue-specific double-stranded RNA molecules that interfere with the expression of a target gene.”


Title: Therapeutic Uses of Inhibitors of RTP801

Application Number: 20130095117

Filed: Aug. 31, 2012

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

The invention comprises “novel molecules, compositions, methods, and uses for treating microvascular disorders, eye diseases, and respiratory conditions based upon inhibition of the RTP801 gene and/or protein,” according to the patent application’s abstract.


Title: Composition for Nucleic Acid Delivery Using Metal Nanoparticles and Preparing Method Thereof

Application Number: 20130095187

Filed: March 27, 2012

Lead Inventor: Sei Kwang Hahn, Postech

The invention, the patent application’s abstract states, “relates to a composition for nucleic acid delivery and a method for preparing the same.”


Title: miRNA-regulated Differentiation-dependent Self-deleting Cassette

Application Number: 20130095565

Filed: Dec. 3, 2012

Lead Inventor: David Frendewey, Regeneron Pharmaceuticals

The patent application, its abstract states, claims “targeting constructs and methods of using them … for differentiation-dependent modification of nucleic acid sequences in cells and in non-human animals. Targeting constructs [comprise] a promoter operably linked to a recombinase [that] … drives transcription of the recombinase in a differentiated cell but not an undifferentiated cell. Promoters include Blimp1, Prm1, Gata6, Gata4, Igf2, Lhx2, Lhx5, and Pax3.

“Targeting constructs with a cassette flanked on both sides by recombinase sites can be removed using a recombinase gene operably linked to a 3′-UTR that comprises a recognition site for an miRNA that is transcribed in undifferentiated cells but not in differentiated cells,” the abstract adds.
“The constructs may be included in targeting vectors, and can be used to automatically modify or excise a selection cassette from an ES cell, a non-human embryo, or a non-human animal.”


Title: Polymers for Delivering Molecules of Interest

Application Number: 20130096177

Filed: March 29, 2011

Lead Inventor: Guy Zuber, National Center for Scientific Research

The invention, the patent application’s abstract states, “relates to a new class of cationic polymers that self-assemble with a pH-sensitive dissolution switch, and their uses to deliver molecules of interest to a cell. The … invention also relates to compositions comprising said cationic polymers non-covalently associated with a molecule of interest, in particular with a siRNA.”


Title: siRNA Conjugates and Preparation Method Thereof

Application Number: 20130096288

Filed: Sept. 13, 2012

Lead Inventor: Bo Ram Han, Bioneer

The patent application, its abstract states, claims a “hybrid conjugate formed by covalently bonding siRNA and a polymeric compound for improving the in vivostability of siRNA, and to a preparation method of the hybrid conjugate. The conjugate … can improve the in vivo stability of siRNA, thereby achieving an efficient delivery of therapeutic siRNA into cells and exhibiting the activity of siRNA even with a small dose of a relative low concentration. Therefore, the conjugate can advantageously be used as not only an siRNA treatment tool for cancers and other infectious disease, but also a novel type siRNA delivery system.”


Title: Single-stranded Extended Dicer-substrate Agents and Methods of the Specific Inhibition of Gene Expression

Application Number: 20130096290

Filed: Dec. 7, 2012

Inventor: Bob Brown, Dicerna Pharmaceuticals

The invention, the patent application’s abstract states, “provides compositions and methods for reducing expression of a target gene in a cell [by] contacting a cell with an isolated double-stranded nucleic acid in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single-stranded extension. Such single-stranded extended Dicer-substrate siRNAs were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.”


Title: Compositions and Systems for the Regulation of Genes

Application Number: 20130096370

Filed: April 17, 2012

Lead Inventor: Didier Trono, Swiss Federal Institute of Technology

The invention comprises “compositions and methods of modulating or regulating eukaryotic gene expression through the controlled or regulated expression of polynucleotide constructs that encode siRNA or other desired exogenous nucleic acids or proteins,” the patent application’s abstract states. “Such constructs, and additional elements of the system may be transfected into the cells of interest and the expression of the siRNA, and hence the expression of the target gene of the siRNA, may be controlled through the administration of a compound to the cell, such as a small molecule or drug. Lentivirus vectors are employed in some embodiments of the invention including the generation of conditional knockdown animals.”


Title: Composition and Method for Prolonging the Shelf Life of Banana by Using RNA Interference

Application Number: 20130097732

Filed: July 6, 2012

Lead Inventor: Pung-Ling Huang, National Taiwan University

“The invention relates to a composition and method for prolonging the shelf life of banana by using interfering RNA,” the patent application’s abstract states. The method involves transferring a control cassette for ACC oxidase into a banana by a novel gene transfer method. The cassette comprises an interfering RNA, a gene transfer expression vector, and pharmaceutically acceptable carrier, and can block ACC oxidase expression to inhibit the biosynthesis of ethylene in a banana to delay ripening.

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Reporter: Aviva Lev-Ari, PhD, RN

 

Harnessing New Players in Atherosclerosis to Treat Heart Disease

Tuesday, September 24, 2013 | 8:30 AM – 4:30 PM

The New York Academy of Sciences

Presented by the Biochemical Pharmacology Discussion Group

Atherosclerosis is defined as a chronic inflammatory disease affecting arterial blood vessels involving dysregulation of the endothelial-leukocyte adhesive interactions, increased leukocyte apoptosis within the plaque, and defective phagocytosis of apoptotic cells. Despite the key role of monocytes/macrophages in atherosclerosis, mounting evidence suggests that dysregulation of other cell types may be independent risk factors for atherosclerosis. Leukocytes are produced daily and are derived from hematopoietic stem and progenitor cells within the bone marrow in a process call hematopoiesis. A better understanding of this process will open an avenue to identify new targets to fight atherosclerosis.

*Reception to follow.

Organizers

Mercedes Beyna, MS

Pfizer Global Research and Development

Nadeem Sarwar, PhD

Pfizer Global Research and Development

Laurent Yvan-Charvet, PhD

INSERM U1065/UNS, C3M

Jennifer Henry, PhD

The New York Academy of Sciences

Speakers

Elena V. Galkina, MD, PhD

Eastern Virginia Medical School

Emmanuel L. Gautier, PhD

Washington University School of Medicine, St. Louis

Klaus Ley, MD

La Jolla Institute for Allergy and Immunology

Andrew H. Lichtman, MD, PhD

Brigham and Women’s Hospital, Harvard Medical School

Kathryn J. Moore, PhD

New York University Medical Center

Matthias Nahrendorf, MD, PhD

Harvard Medical School

Alan R. Tall, MD, PhD

Columbia University Medical Center

http://www.nyas.org/Events/Detail.aspx?cid=1103f191-2d94-4f37-b91f-64293dc88019

 

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Revascularization: PCI, Prior History of PCI vs CABG

Curator: Aviva Lev-Ari, PhD, RN

 

UPDATED 9/25/2013

Table. Comparison of Surgical Therapy and Coronary Angioplasty (Open Table in a new window)

Endpoint Pocock et al* Pocock et al BARI Study
CABG(N=358) PTCA(N=374) CABG(N=1303) PTCA(N=1336) CABG(N=914) PTCA(N=915)
Death (%) 0.3 1.9 2.8 3.1 10.7 13.7
Death or MI 4.5 7.2 8.5 8.1 11.7 10.9
Repeat CABG 1.4 16.0§ 0.8 18.3§ 0.7 20.5§
Repeat CABG or PTCA 3.6 30.5§ 3.2 34.5§ 8.0 54.0§
More than mild angina 6.5 14.6§ 12.1 17.8§
*Meta-analysis of results of 3 trials at 1 year. Patients with single-vessel disease were studied.[22] †Meta-analysis of results of 3 trials at 1 year. Patients with multivessel disease were studied.[22] 

‡Reported results are for 5-year follow-up. Patients with multivessel disease were studied.[21] 

§ P < .05.

BARI = Bypass Angioplasty Revascularization Investigation; CABG = coronary artery bypass grafting; MI = myocardial infarction; PTCA = percutaneous transluminal coronary angioplasty.

SOURCE

http://emedicine.medscape.com/article/161446-overview#aw2aab6b2b5

Percutaneous coronary intervention (PCI), also known as coronary angioplasty, is a nonsurgical technique for treating multiple conditions, including unstable angina, acute myocardial infarction (MI), and multivessel coronary artery disease (CAD).

Essential update: Cangrelor decreases periprocedural complications of PCI

According to a pooled analysis of 3 CHAMPION trials—CHAMPION-PCI , CHAMPION-PLATFORM , and CHAMPION-PHOENIX—cangrelor can reduce the risk of periprocedural thrombotic complications of PCI.[1, 2, 3] The 3 trials included patients with ST-elevation MI (STEMI), non-STEMI, and stable CAD who were randomly assigned to receive either cangrelor or control therapy consisting of either clopidogrel or placebo.

The primary outcome in this analysis was a composite of death, MI, ischemia-driven revascularization, or stent thrombosis at 48 hours.[2] The frequency of this outcome was significantly lower in cangrelor-treated patients than in control subjects (absolute difference, 1.9%; relative risk reduction [RRR], 19%). Stent thrombosis was also reduced in the cangrelor-treated group (absolute difference, 0.3%; RRR, 41%). Primary safety outcomes were comparable in the 2 groups, but cangrelor-treated patients had a higher rate of mild bleeding.

Indications and contraindications

Clinical indications for PCI include the following:

In an asymptomatic or mildly symptomatic patient, objective evidence of a moderate-sized to large area of viable myocardium or moderate to severe ischemia on noninvasive testing is an indication for PCI. Angiographic indications include hemodynamically significant lesions in vessels serving viable myocardium (vessel diameter >1.5 mm).

Clinical contraindications for PCI include the presence of any significant comorbid conditions (this is a relative contraindication). Angiographic contraindications include the following:

  • Left main stenosis in a patient who is a surgical candidate (except in carefully selected patients[4] )
  • Diffusely diseased small-caliber artery or vein graft
  • Other coronary anatomy not amenable to PCI

In patients with stable angina, medical therapy is recommended as first-line therapy unless one or more of the following indications for cardiac catheterization and PCI or CABG are present:

  • A change in symptom severity
  • Failed medical therapy
  • High-risk coronary anatomy
  • Worsening left ventricular (LV) dysfunction

American College of Cardiology Foundation/American Heart Association (ACCF/AHA) guidelines on the management of unstable angina/non-STEMI recommend that an early invasive approach (angiography and revascularization within 24 hours) should be used to treat patients presenting with the following high-risk features[5] :

  • Recurrent angina at rest or low level of activity
  • Elevated cardiac biomarkers
  • PCI in the past 6 months or prior CABG
  • New ST-segment depression
  • Elevated cardiac biomarkers
  • High-risk findings on noninvasive testing
  • Signs or symptoms of heart failure or new or worsening mitral regurgitation
  • Hemodynamic instability
  • Sustained ventricular tachycardia
  • LV systolic function < 40%
  • High risk score (eg, Thrombolysis in Myocardial Infarction [TIMI] score >2) (see the TIMI Score for Unstable Angina Non ST Elevation Myocardial Infarction calculator)

See Overview for more detail.

Equipment

Balloon catheters for PCI have the following features:

  • A steerable guide wire precedes the balloon into the artery and permits navigation through the coronary tree
  • Inflation of the balloon compresses and axially redistributes atheromatous plaque and stretches the vessel wall
  • The balloon catheter also serves as an adjunctive device for many other interventional therapies

Atherectomy devices have the following features:

  • These devices are designed to physically remove coronary atheroma, calcium, and excess cellular material
  • Rotational atherectomy, which relies on plaque abrasion and pulverization, is used mostly for fibrotic or heavily calcified lesions that can be wired but not crossed or dilated by a balloon catheter
  • Atherectomy devices may be used to facilitate stent delivery in complex lesions
  • Directional coronary atherectomy (DCA) has been used to debulk coronary plaques
  • Laser atherectomy is not widely used at present
  • Atherectomy is typically followed by balloon dilation and stenting

Intracoronary stents have the following features:

  • Stents differ with respect to composition (eg, stainless steel, cobalt chromium, or nickel chromium), architectural design, and delivery system
  • Drug-eluting stents have demonstrated significant reductions in restenosis and target-lesion revascularization rates
  • In the United States, stents are available that elute the following drugs: sirolimus (Cypher), paclitaxel (Taxus), zotarolimus (Endeavor), and everolimus (Xience V)
  • Stents are conventionally placed after balloon predilation, but in selected coronary lesions, direct stenting may lead to better outcomes

Other devices used for PCI include the following:

  • Thrombus aspiration limits the adverse effects that prolonged time to treatment has on myocardial reperfusion[6]
  • Distal embolic protection during saphenous vein graft intervention has become the standard of care

See Periprocedural Care and Devices for more detail.

Technique

Intravascular ultrasonography (IVUS) is used in PCI as follows:

  • Provide information about the plaque, the vessel wall, and the degree of luminal narrowing
  • Assessment of indeterminate lesions
  • Evaluation of adequate stent deployment

Intracoronary Doppler pressure wires are used in PCI as follows:

  • To characterize coronary lesion physiology and estimate lesion severity
  • Comparison of pressure distal to a lesion with aortic pressure enables determination of fractional flow reserve (FFR)
  • An FFR measurement below 0.75-0.80 during maximal hyperemia (induced via administration of adenosine) is consistent with a hemodynamically significant lesion

Antithrombotic therapy

  • Aspirin and heparin have been the traditional adjunctive medical therapies
  • Direct thrombin inhibitors (ie, hirudin, bivalirudin) are slightly better than heparin in preventing ischemic complications during balloon angioplasty but do not affect restenosis rates
  • Low-molecular-weight heparins (LMWHs) are substituted for standard heparin at some centers

Antiplatelet therapy

Patients receiving stents are treated with a combination of aspirin and clopidogrel. Duration of therapy is as follows:

  • Bare-metal stents: A minimum of 4 weeks
  • Drug-eluting stents: A minimum of 12 months

Use of proton pump inhibitors is appropriate in patients with multiple risk factors for GI bleeding who require antiplatelet therapy.

Glycoprotein inhibitor therapy

  • Abciximab, tirofiban, and eptifibatide have all been shown to reduce ischemic complications in patients undergoing balloon angioplasty and coronary stenting
  • In primary PCI, GPIIb/IIIa receptor inhibitors have also been shown to improve flow and perfusion and to reduce adverse events
  • Abciximab may improve outcomes in patients when given before arrival in the catheterization lab for primary PCI[7]

See Technique and Medication for more detail.

SOURCE & References for the UPDATE, in

http://emedicine.medscape.com/article/161446-overview#aw2aab6b2b5

Outcomes comparison between PCI and CABG was explored in the past by authors on this Open Access Online Scientific Journal, in the following articles:

CABG or PCI: Patients with Diabetes – CABG Rein Supreme

http://pharmaceuticalintelligence.com/2012/11/05/cabg-or-pci-patients-with-diabetes-cabg-rein-supreme/

To Stent or Not? A Critical Decision

http://pharmaceuticalintelligence.com/2012/10/23/to-stent-or-not-a-critical-decision/

PCI Outcomes, Increased Ischemic Risk associated with Elevated Plasma Fibrinogen not Platelet Reactivity

http://pharmaceuticalintelligence.com/2013/01/10/pci-outcomes-increased-ischemic-risk-associated-with-elevated-plasma-fibrinogen-not-platelet-reactivity/

New Definition of MI Unveiled, Fractional Flow Reserve (FFR)CT for Tagging Ischemia

http://pharmaceuticalintelligence.com/2012/08/27/new-definition-of-mi-unveiled-fractional-flow-reserve-ffrct-for-tagging-ischemia/

Age-Dependent Depression in Circulating Endothelial Progenitor Cells in Coronary Artery Bypass Grafting Patients

http://pharmaceuticalintelligence.com/2012/08/17/age-dependent-depression-in-circulating-endothelial-progenitor-cells-in-coronary-artery-bypass-grafting-patients/

Now we are reporting  an Original Contribution on this subject which includes also Prior History of PCI, a factor NOT included in the other studies. The major conclusions are the following three:

  1. In a contemporary cohort of STEMI patients undergoing primary PCI, a history of prior CABG was found to be an independent predictor of in-hospital mortality.
  2. In contrast, despite more comorbidities at the time of STEMI, patients with prior PCI had no significant difference in the rates of death, stroke, or periprocedural MI when compared to a STEMI population without prior coronary revascularization.
  3. Thus, only prior surgical — and not percutaneousrevascularization should be considered a significant risk factor in the setting of primary PCI.

Number 1, above is related to patient medical history of cardiovascular disease SEVERITY prior to CABG

Number 2, above indicates that patients can tolerate and benefit several cycles of PCI and stent implantation rather than PCI being a determinant predictor of future prognosis

Number 3, above is as well related to patient medical history of cardiovascular disease SEVERITY prior to CABG

The Original Contribution on this subject is present, below.

The Impact of Previous Revascularization on Clinical Outcomes in Patients Undergoing Primary Percutaneous Coronary Intervention

Travis J. Bench, MD1, Puja B. Parikh, MD1, Allen Jeremias, MD1, Sorin J. Brener, MD2, Srihari S. Naidu, MD3,

Richard A. Shlofmitz, MD4, Thomas Pappas, MD4, Kevin P. Marzo, MD3, Luis Gruberg, MD1

Authors Affiliations:

1Division of Cardiovascular Medicine, Stony Brook University Medical Center, Stony Brook, New York,

2Department of Cardiology, Methodist Hospital, Brooklyn, New York,

3Division of Cardiology, Winthrop University Hospital, Mineola,

New York, and

4The Heart Center, St Francis Hospital, Roslyn, New York.

The authors report no conflicts of interest regarding the content herein.

Manuscript submitted October 10, 2012, provisional acceptance given October 20, 2012, final version accepted November 28, 2012.

Address for correspondence: 

Luis Gruberg, MD, FACC, Department of Medicine, Division of Cardiology, Health Sciences Center, T16-080, Stony Brook, NY 11794- 8160. Email: luis.gruberg@stonybrook.edu

 

Abstract : While the impact of prior coronary artery bypass graft surgery (CABG) on in-hospital outcomes in patients with STelevation myocardial infarction (STEMI) has been described, data are limited on patients with prior percutaneous coronary intervention (PCI) undergoing primary PCI in the setting of an STEMI. The aim of the present study was to assess the effect of previous revascularization on in-hospital outcomes in STEMI patients undergoing primary PCI. Between January 2004 and December 2007, a total of 1649 patients underwent primary PCI for STEMI at four New York State hospitals. Baseline clinical and angiographic characteristics and in-hospital outcomes were prospectively collected as part of the New York State PCI Reporting System (PCIRS). Patients with prior surgical or percutaneous coronary revascularization were compared to those without prior coronary revascularization. Of the 1649 patients presenting with STEMI, a total of 93 (5.6%) had prior CABG, 258 (15.7%) had prior PCI, and 1298 (78.7%) had no history of prior coronary revascularization. Patients with prior CABG were significantly older and had higher rates of peripheral vascular disease, diabetes mellitus, congestive heart failure, and prior stroke. Additionally, compared with those patients with a history of prior PCI as well as those without prior coronary revascularization, patients with previous CABG had more left main interventions (24% vs 2% and 2%; P<.001), but were less often treated with drug-eluting stents (47% vs 61% and 72%; P<.001).

Despite a low incidence of adverse in-hospital events, prior CABG was associated with higher all-cause in-hospital mortality (6.5% vs 2.2%; P=.012), and as a result, higher overall MACE (6.5% vs 2.7%; P=.039). By multivariate analysis, prior CABG (odds ratio, 3.40; 95% confidence interval, 1.15-10.00) was independently associated with in-hospital mortality. In contrast, patients with prior PCI had similar rates of MACE (4.3% vs 2.7%; P=.18) and inhospital mortality (3.1% vs 2.2%; P=.4) when compared to the de novo population. Patients with a prior history of CABG, but not prior PCI, undergoing primary PCI in the setting of STEMI have significantly worse in-hospital outcomes when compared with patients who had no prior history of coronary artery revascularization. Thus, only prior surgical — and not percutaneous — revascularization should be considered a significant risk factor in the setting of primary PCI.

J INVASIVE CARDIOL 2013;25(4):166-169

Key words: PCI risk factor, CABG

Demographics and Angiographic Characteristics

Between 2004 and 2007, a total of 25,025 patients underwent PCI at these medical institutions, and their data were prospectively collected and submitted as required by the New York State Department of Health. Of these patients, a total of 1649 underwent primary PCI in the setting of an STEMI and constituted our study population. In this group, a total

No Prior Revascularization (n = 1298)

Prior PCI (n = 258)

Prior CABG (n = 93)

Demographics

Age (years) 61 ± 13 62 ± 12 67 ± 12 <.001

Male gender 956 (73.6%) 194 (75.2%) 76 (81.7%) .21

White 1165 (89.8%) 231 (89.5%) 87 (93.5%) .51

African-American 78 (6%) 18 (7%) 1 (1.1%) .51

Hispanic 91 (7%) 11 (4.3%) 4 (4.3%) .51

Medical history

Ejection fraction (%) 43 ± 12 44 ± 13 45 ± 11 .079

Diabetes mellitus 196 (15.1%) 69 (26.7%) 27 (29%) <.001

Peripheral vascular disease 53 (4.1%) 25 (9.7%) 12 (12.9%) <.001

Chronic lung disease 47 (3.6%) 17 (6.6%) 4 (4.3%) .09

Congestive heart failure 74 (5.7%) 25 (9.7%) 10 (10.8%) .02

Prior myocardial infarction 3 (0.2%) 1 (0.4%) 1 (1.1%) .35

Prior cerebrovascular event 56 (4.3%) 9 (3.5%) 10 (11%) .01

Chronic dialysis 6 (0.5%) 6 (2.3%) 0 (0%) .004

Creatinine (mg/dL) 1.1 ± 0.8 1.3 ± 1.4 1.3 ± 1.1 .002

Glomerular filtration rate (mL/min/1.73 m2) 79 ± 26 75 ± 28 71 ± 27 .002

Angiographic characteristics

Left main 19 (1.5%) 5 (1.9%) 22 (23.7%) <.001

Left anterior descending 942 (72.6%) 178 (69%) 69 (74.2%) .45

Left circumflex 579 (44.6%) 122 (47.3%) 70 (75.3%) <.001

Right coronary 806 (62.1%) 187 (72.5%) 67 (72%) .002

Graft (arterial or venous) n/a n/a 20 (21.5%)

Stent type

Bare-metal stent 241 (18.6%) 52 (20.2%) 23 (24.7%) .31

Drug-eluting stent 928 (71.5%) 158 (61.2%) 44 (47.3%) <.001

of 1298 patients (78.7%) had no prior history of revascularization,

while 93 patients (5.6%) had a history of previous

CABG and 258 (15.7%) had a history of previous PCI. Considerable

differences in baseline clinical and procedural characteristics were noted among these groups (Table 1).

Discussion

While STEMI patients with prior CABG are well known to have worse clinical outcomes than those without prior revascularization, a direct comparison between patients who underwent primary PCI in the setting of prior CABG or prior PCI has not yet been reported. The principal findings from the present analysis suggest that in a contemporary, unrestricted patient population presenting with STEMI and undergoing primary PCI, patients with a prior history of CABG are:

(1) usually older and have multiple comorbidities, including peripheral vascular disease, diabetes, and chronic obstructive lung disease;

(2) are more likely to undergo intervention on a native vessel and not a bypass graft;

(3) are more likely to be treated with bare-metal stents; and (4) have higher rates of in-hospital mortality without a significant increase in stroke or MI rates, when compared with patients with a prior history of PCI or patients with no previous history of coronary artery revascularization. Interestingly, these outcomes did not apply to patients with a history of prior PCI in this analysis. Instead, this cohort of patients had no significant difference in the rate of death, stroke, or periprocedural infarction when compared to a STEMI population without prior coronary revascularization, despite a significantly higher burden of comorbidities than those with no prior revascularization.

Our findings concur with previous studies that have shown higher mortality rates among patients with prior surgical bypass presenting with acute MI.7,9,14 Despite changes in revascularization strategies over the past 30 years, invasive therapies to treat acute coronary syndromes in patients with prior bypass surgery appear to have yielded less robust results than in other populations. In fact, Stone and colleagues already described in the Primary Angioplasty in Myocardial Infarction (PAMI-2) study that patients with a previous CABG undergoing primary PCI in the setting of an acute MI had significantly greater in-hospital mortality than patients without previous CABG, especially if the infarct-related vessel was a bypass conduit. However, by logistic regression analysis, only advanced age (P=.004), triple-vessel disease (P=.004), and Killip class ≥2 (P=.02) were independent predictors of in-hospital mortality in that study.13 In a more contemporary study of 128 STEMI patients with prior CABG, who were enrolled in the Assessment of PEXelizumab in Acute

Figure 1. In-hospital major adverse cardiac and cerebrovascular events (MACCE), mortality, and stroke rates for patients without prior history of coronary revascularization (light grey bars), prior percutaneous coronary revascularization (PCI) (dark grey bars), and prior coronary artery bypass graft (CABG) (black bars). Vol. 25, No. 4, April 2013 169

STEMI and Prior Revascularization Myocardial Infarction (APEX-AMI) trial, Welsh and colleagues reported that post-CABG patients are less likely to undergo acute reperfusion (only 79% underwent primary PCI), have worse angiographic outcomes following primary PCI, and have higher 90-day mortality rates (19.0% vs 5.7%; P=.05). This difference was even more apparent when the infarct-related artery was a bypass graft that was not successfully reperfused (23.1% vs 8.5%; P=.03).3 These results are similar to our current analysis, where in-hospital mortality rates for patients who underwent primary PCI of a graft were numerically roughly 4 times as high as those undergoing PCI of a native vessel. Likewise, Gurfinkel et al reported a significant reduction in hard endpoints, such as all-cause death and MI at 6 months in patients treated with an invasive approach in the Global Registry of Acute Coronary Events (GRACE).15 In this large, multinational, observational study of 3853 patients with prior bypass surgery presenting with an acute coronary syndrome, only 497 (12.9%) were managed invasively and the rest were treated medically.

Despite significant differences in baseline characteristics, including a higher rate of STEMI in patients treated invasively (14% vs 27%; P<.001), in-hospital mortality was similar in both groups (3.4% vs 3.2%; P=.86). However, at 6-month follow-up, mortality was significantly higher in those patients treated medically (6.5% vs 3.4%; P<.02) as was the combined endpoint of death or MI (11% vs 5.8%; P<.01).

Whether these results apply to patients with a prior history of PCI has not been well defined. By the nature of vascular disease, patients with prior PCI are more likely to have more comorbidities than those without prior revascularization, a finding confirmed in our study. Despite considerable differences in baseline characteristics, however, these differences did not translate into a differential risk after STEMI. In fact, the cohort of patients presenting with STEMI who had a history of prior PCI had no statistically significant difference in in-hospital mortality or overall MACCE when compared to a population of patients presenting with STEMI in the absence of any prior revascularization.

Study limitations. The database utilized was derived from four New York State teaching hospitals and was designed to track quality of care and clinical outcomes. As all studies involving multicenter databases and registries, there is potential error in data entry and availability. Potential confounding comorbidities, including smoking status and family history of coronary artery disease, were not collected in this database, and information regarding long-term follow-up is not available, all of which are important limitations of this analysis. As such, deficiencies such as these limit the conclusions that can be drawn from our multivariate analysis. Additionally, there is no audit of data quality, and the low overall event rates limit effective statistical comparison.

Conclusions

In a contemporary cohort of STEMI patients undergoing primary PCI, a history of prior CABG was found to be an independent predictor of in-hospital mortality. In contrast, despite more comorbidities at the time of STEMI, patients with prior PCI had no significant difference in the rates of death, stroke, or periprocedural MI when compared to a STEMI population without prior coronary revascularization. Thus, only prior surgical — and not percutaneous — revascularization should be considered a significant risk factor in the setting of primary PCI.

REFERENCES

1. Kushner FG, Hand M, Smith SC Jr, et al. 2009 focused updates: ACC/AHA guidelines for the management of patients with ST-elevation myocardial infarction (updating the 2004 guideline and 2007 focused update) and ACC/AHA/SCAI guidelines on percutaneous coronary intervention (updating the 2005 guideline and 2007 focused update): a report of the American College of Cardiology Foundation/American Heart Association Task Force on Practice Guidelines. Catheter Cardiovasc Interv. 2009;74(7):E25-E68.

2. Keeley EC, Boura JA, Grines CL. Primary angioplasty versus intravenous thrombolytic therapy for acute myocardial infarction: a quantitative review of 23 randomised trials. Lancet. 2003;361(9351):13-20.

3. Welsh RC, Granger CB, Westerhout CM, et al. Prior coronary artery bypass graft patients with ST-segment elevation myocardial infarction treated with primary percutaneous coronary intervention. JACC Cardiovasc Interv. 2010;3(3):343-351.

4. Mathew V, Gersh B, Barron H, et al. In-hospital outcome of acute myocardial infarction in patients with prior coronary artery bypass surgery. Am Heart J. 2002;144(3):463-469.

5. Lee KL, Woodlief LH, Topol EJ, et al. Predictors of 30-day mortality in the era of reperfusion for acute myocardial infarction. Results from an international trial of 41,021 patients. GUSTO-I Investigators. Circulation. 1995;91(6):1659-1668.

6. Dittrich HC, Gilpin E, Nicod P, et al. Outcome after acute myocardial infarction in patients with prior coronary artery bypass surgery. Am J Cardiol. 1993;72(7):507-513.

7. Berry C, Pieper KS, White HD, et al. Patients with prior coronary artery bypass grafting have a poor outcome after myocardial infarction: an analysis of the VALsartan in acute myocardial iNfarcTion trial (VALIANT). Eur Heart J. 2009;30(12):1450-1456.

8. Grines CL, Booth DC, Nissen SE, et al. Mechanism of acute myocardial infarction in patients with prior coronary artery bypass grafting and therapeutic implications. Am J Cardiol. 1990;65(20):1292-1296.

9. Labinaz M, Sketch MH Jr, Ellis SG, et al. Outcome of acute ST-segment elevation myocardial infarction in patients with prior coronary artery bypass surgery receiving thrombolytic therapy. Am Heart J. 2001;141(3):469-477.

10. Peterson LR, Chandra NC, French WJ, Rogers WJ, Weaver WD, Tiefenbrunn AJ. Reperfusion therapy in patients with acute myocardial infarction and prior coronary artery bypass graft surgery (National Registry of Myocardial Infarction-2). Am J Cardiol. 1999;84(11):1287-1291.

11. Nguyen TT, O’Neill WW, Grines CL, et al. One-year survival in patients with acute myocardial infarction and a saphenous vein graft culprit treated with primary angioplasty. Am J Cardiol. 2003;91(10):1250-1254.

12. Al Suwaidi J, Velianou JL, Berger PB, et al. Primary percutaneous coronary interventions in patients with acute myocardial infarction and prior coronary artery bypass grafting, Am Heart J. 2001;142(3):452-459.

13. Stone GW, Brodie BR, Griffin JJ, et al. Clinical and angiographic outcomes in patients with previous coronary artery bypass graft surgery treated with primary balloon angioplasty for acute myocardial infarction. Second Primary Angioplasty in Myocardial Infarction Trial (PAMI-2) Investigators. J Am Coll Cardiol. 2000;35(3):605-611.

14. Labinaz M, Kilaru R, Pieper K, et al. Outcomes of patients with acute coronary syndromes and prior coronary artery bypass grafting: results from the platelet glycoprotein IIb/IIIa in unstable angina: receptor suppression using integrilin therapy (PURSUIT) trial. Circulation. 2002;105(3):322-327.

15. Gurfinkel EP, Perez de la Hoz R, Brito VM, et al. Invasive vs non-invasive treatment in acute coronary syndromes and prior bypass surgery. Int J Cardiol. 2007;119(1):65-72.

 

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Risk of a Second Primary Cancer after Non-melanoma Skin Cancer in White Men and Women: A Prospective Cohort Study

  • Fengju Song,
  • Abrar A. Qureshi,
  • Edward L. Giovannucci,
  • Charlie S. Fuchs,
  • Wendy Y. Chen,
  • Meir J. Stampfer,
  • Jiali Han mail
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Abstract

Background

Previous studies suggest a positive association between history of non-melanoma skin cancer (NMSC) and risk of subsequent cancer at other sites. The purpose of this study is to prospectively examine the risk of primary cancer according to personal history of NMSC.

Methods and Findings

In two large US cohorts, the Health Professionals Follow-up Study (HPFS) and the Nurses’ Health Study (NHS), we prospectively investigated this association in self-identified white men and women. In the HPFS, we followed 46,237 men from June 1986 to June 2008 (833,496 person-years). In the NHS, we followed 107,339 women from June 1984 to June 2008 (2,116,178 person-years). We documented 29,447 incident cancer cases other than NMSC. Cox proportional hazard models were used to calculate relative risks (RRs) and 95% confidence intervals (CIs). A personal history of NMSC was significantly associated with a higher risk of other primary cancers excluding melanoma in men (RR = 1.11; 95% CI 1.05–1.18), and in women (RR = 1.20; 95% CI 1.15–1.25). Age-standardized absolute risk (AR) was 176 in men and 182 in women per 100,000 person-years. For individual cancer sites, after the Bonferroni correction for multiple comparisons (n = 28), in men, a personal history of NMSC was significantly associated with an increased risk of melanoma (RR = 1.99, AR = 116 per 100,000 person-years). In women, a personal history of NMSC was significantly associated with an increased risk of breast (RR = 1.19, AR = 87 per 100,000 person-years), lung (RR = 1.32, AR = 22 per 100,000 person-years), and melanoma (RR = 2.58, AR = 79 per 100,000 person-years).

Conclusion

This prospective study found a modestly increased risk of subsequent malignancies among individuals with a history of NMSC, specifically breast and lung cancer in women and melanoma in both men and women.

Please see later in the article for the Editors’ Summary

Editors’ Summary

Background

In the United Kingdom and the United States, about one in three people develop cancer during their lifetime and, worldwide, cancer is responsible for 13% of all deaths. Primary cancer, which can develop anywhere in the body, occurs when a cell begins to divide uncontrollably because of alterations (mutations) in its genes. Additional mutations allow the malignancy to spread around the body (metastasize) and form secondary cancers. The mutations that initiate cancer can be triggered by exposure to carcinogens such as cigarette smoke (lung cancer) or the ultraviolet (UV) radiation in sunlight (skin cancers). Other risk factors for the development of cancer include an unhealthy diet, physical inactivity, and alcohol use. In the United States, the most common cancer is non-melanoma skin cancer (NMSC). Although more than 2 million new cases of NMSC occur each year, fewer than 1,000 people die annually in the United States from the condition because the two types of NMSC—basal cell carcinoma and squamous cell carcinoma—rarely metastasize and can usually be treated by surgically removing the tumor.

Why Was This Study Done?

Some studies have suggested that people who have had NMSC have a higher risk of developing primary cancer at other sites than people who have not had NMSC. Such a situation could arise if exposure to certain carcinogens initiates both NMSC and other cancers or if NMSC shares a molecular mechanism with other cancers such as a deficiency in the DNA repair mechanisms that normally remove mutations. If people with a history of NMSC are at a greater risk of developing further cancers, a specific surveillance program for such people might help to catch subsequent cancers early when they can be successfully treated. In this prospective cohort study, the researchers examine the risk of primary cancer according to personal history of NMSC in two large US cohorts (groups)—the Health Professionals Follow-up Study (HPFS) and the Nurses’ Health Study (NHS). The HPFS, which enrolled 51,529 male health professionals in 1986, and the NHS, which enrolled 121,700 female nurses in 1976, were both designed to investigate associations between nutritional factors and the incidence of serious illnesses. Study participants completed a baseline questionnaire about their lifestyle, diet and medical history. This information is updated biennially through follow-up questionnaires.

What Did the Researchers Do and Find?

The researchers identified 36,102 new cases of NMSC and 29,447 new cases of other primary cancers from 1984 in white NHS participants and from 1986 in white HPFS participants through 2008. They then used statistical models to investigate whether a personal history of NMSC was associated with a higher risk of subsequent primary cancers after accounting for other factors (confounders) that might affect cancer risk. A history of NMSC was significantly associated with an 11% higher risk of other primary cancers excluding melanoma (another type of skin cancer that, like NMSC, is linked to overexposure to UV light) in men and a 20% higher risk of other primary cancers excluding melanoma in women; a significant association is one that is unlikely to have happened by chance. The absolute risk of a primary cancer among men and women with a history of NMSC was 176 and 182 per 100,000 person-years, respectively. For individual cancer sites, after correction for multiple comparisons (when several conditions are compared in groups of people, statistically significant differences between the groups can occur by chance), a history of NMSC was significantly associated with an increased risk of breast and lung cancer in women and of melanoma in men and women.

What Do These Findings Mean?

These findings suggest that there is a modestly increased risk of subsequent malignancies among white individuals with a history of NMSC. Although the researchers adjusted for many confounding lifestyle factors, the observed association between NMSC and subsequent primary cancers may nevertheless be the result of residual confounding, so it is still difficult to be sure that there is a real biological association (due to, for example, a deficiency in DNA repair) between NMSC and subsequent primary cancers. Because of this and other study limitations, the findings reported here should be interpreted cautiously and do not suggest that individuals who have had NMSC should undergo increased cancer surveillance. These findings do, however, support the need for continued investigation of the apparent relationship between NMSC and subsequent cancers.

Additional Information

Please access these Web sites via the online version of this summary athttp://dx.doi.org/10.1371/journal.pmed.1​001433.

Citation: Song F, Qureshi AA, Giovannucci EL, Fuchs CS, Chen WY, et al. (2013) Risk of a Second Primary Cancer after Non-melanoma Skin Cancer in White Men and Women: A Prospective Cohort Study. PLoS Med 10(4): e1001433. doi:10.1371/journal.pmed.1001433

Academic Editor: Eduardo L. Franco, McGill University, Canada

Received: September 11, 2012; Accepted: March 15, 2013; Published: April 23, 2013

Copyright: © 2013 Song et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

Funding: This work was supported by US NIH CA87969 and CA055075. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.

Competing interests: AQ declares the following: Pfizer – Questionnaire licensed for clinical trials; research grant. Merck – Questionnaire licensed for clinical trials. Amgen – Research grant. Abbott – Consulting. US Centers for Disease Control – Consulting. Janssen – Consulting. Novartis – Consulting. All other authors have declared that no competing interests exist.

Abbreviations: AR, absolute risk; BCC, basal cell carcinoma; BMI, body mass index; HPFS, Health Professionals Follow-up Study; MV, multivariate; NER, nucleotide excision repair; NHS, Nurses’ Health Study; NMSC, non-melanoma skin cancer; RR, relative risk; SCC, squamous cell carcinoma; UV, ultraviolet

Introduction

Non-melanoma skin cancer (NMSC) is the most common cancer in the United States. It consists mainly of basal cell carcinoma (BCC) and squamous cell carcinoma (SCC). Its incidence has been rapidly increasing over the past several decades and the incidence rate was about 6,000/100,000 in the year 2006 [1]. NMSC has a low mortality rate of 1/100,000 [2], but its high prevalence and the expense of related treatment make NMSC a major public health problem and place it among the costliest cancers in the United States [3]. Individuals with personal history of NMSC may be at an altered risk for developing other primary cancers[4][11]. One view is that sunlight causes NMSC but also produces vitamin D, which in turn may reduce the risk of other cancers [12]. Another view is that NMSC and other cancers may share common carcinogenic exposures or molecular mechanisms in their etiology, such as DNA repair deficiency and immune suppression, and thus the history of NMSC may indicate an increased risk of subsequent cancer development.

Previous studies suggest a positive association between personal history of NMSC and risk of subsequent cancer at other sites [4][11]. Most previous reports, however, were based on cancer registry data without adjustment for potential confounding lifestyle factors [4][10]. The only cohort study was limited by its sample size and lacked adequate power to assess individual cancer sites [11]. We carried out a cohort analysis to evaluate the association between personal history of NMSC and subsequent malignancy in the Nurses’ Health Study (NHS) and the Health Professionals’ Follow-up Study (HPFS).

Methods

Ethics Statement

Our study was approved by the Human Research Committee at the Brigham and Women’s Hospital with written informed consent from all participants.

Study Population

The NHS was established in 1976, when 121,700 registered nurses aged 30–55 y in 11 US states responded to a baseline questionnaire regarding risk factors for cancer. Participants completed self-administered, mailed follow-up questionnaires biennially with updated information on their lifestyle, diet, and medical history. The HPFS began in 1986 when 51,529 US male health professionals, including dentists, veterinarians, pharmacists, and optometrists aged 40–75 y, completed a baseline questionnaire on lifestyle, diet, and newly diagnosed diseases. The information was updated biennially with follow-up questionnaires. The follow-up rates of the participants in both cohorts exceed 90%. These studies were approved by the Human Research Committee at Brigham and Women’s Hospital. Race was self-identified in this study as White, Asian, African American, and others. Only white participants were included in this study, accounting for 95.6% of the total population in the two cohorts. The rationale for focusing the primary hypothesis on white participants only was that the patterns of incidence (and likely the risk factors) for NMSC differ widely by race.

Identification of NMSC and Other Primary Cancers

We have routinely identified cases of NMSC and other primary cancers in both cohorts (from 1984 in the NHS and from 1986 in the HPFS). Participants reported new diagnoses biennially. With their permission, participants’ medical records were obtained and reviewed by physicians to confirm their self-reported diagnosis. Medical records were not obtained for self-reported cases of BCC, because the validity of BCC self-reports was more than 90% in validation studies in our cohorts in early years [13],[14]. The personal history of pathologically confirmed invasive SCC and self-reported BCC was the exposure in this analysis. The study outcome was the occurrence of the first confirmed primary cancer other than NMSC. All other cancer cases were documented by medical records or death certificates, and only confirmed cases were included in the analysis.

Assessment of Covariates

Covariates in this analysis included age (continuous variable), body mass index (BMI) (<21, 21–23, 23–25, 25–27, 27–29, 29–31, >31), physical activity (quintiles), smoking status (never, past 1–14 cigarettes per day, past 15+ cigarettes per day, current 1–14 cigarettes per day, current 15+ cigarettes per day), multi-vitamin use (yes or no), menopause status and hormone replacement therapy use in women (pre-menopause, post-menopause non-user, post-menopause past user, and post-menopause current user), and physical examination in the last 2 y (yes or no). We asked about the location of residence (US states) at birth and at age of 15 and 30. The 50 states (and the District of Columbia) were divided into three ultraviolet (UV) index groups: 5 or less (low UV index); 6 (medium UV index); and 7 or more (high UV index)[15]. We defined participants in these three groups if they resided in the same UV-index region at birth, age of 15 and 30.

Statistical Analysis

Follow-up began in 1984 for the NHS and 1986 for the HPFS when the diagnosis of NMSC was first routinely collected, and follow-up ended in 2008 for both cohorts. Participants who reported a history of cancer (including NMSC) prior to baseline were excluded. Participants contributed person-time from the date of return of the baseline questionnaire (1984 in NHS and 1986 in HPFS) until date of diagnosis of confirmed primary cancer, date of death, or the end of follow-up (May 31, 2008), whichever came first. For those who were lost to follow-up, we censored them at the return date of the last questionnaire. Cox regression analysis with time-dependent covariates was used to determine the relative risks (RRs) and 95% CIs of second primary malignancies associated with a previous NMSC diagnosis. We calculated age-standardized absolute risks (ARs) of second primary malignancies associated with a previous NMSC diagnosis. NMSC diagnosis could change during the follow-up period. For individuals with no personal history of cancer at baseline who went on to be diagnosed with NMSC as a first cancer diagnosis during follow-up, the follow-up period before the NMSC diagnosis contributed person-time to the non-exposure group, and the follow-up period after the NMSC diagnosis contributed person-time to the exposure group. Age was coded as a continuous variable in all the analyses. We showed overall cancer risk with and without melanoma. We performed several secondary analyses. We excluded those diagnosed with other primary cancers within the first 4 y of NMSC diagnosis to minimize the detection bias. We examined BCC and SCC history separately. We performed stratified analysis according to age (≤60 y, >60 y), UV-index of residence at birth, age 15, and age 30 (≤5, = 6, ≥7), smoking status (never smoker, past smoker, current smoker), and BMI (<25, 25–30, ≥30). We coded these factors as dummy variables and tested their interactions with the history of NMSC individually. We tested multiplicative interaction terms by the likelihood ratio test comparing the model with the cross-product terms with the model containing just the main effects of these factors and the history of NMSC along with the same covariates.

We assessed the association between NMSC diagnosis and risk of developing site-specific cancers that were diagnosed in more than 100 patients in each cohort. For individual cancer sites, the Cox models additionally included risk factors specific for some cancer sites. We included additional covariates in the multivariate models for breast, ovarian, endometrial, prostate cancers, and melanoma. The Bonferroni correction for p-value was applied for multiple comparisons for individual cancer sites among men and women, calculated as 0.05/n (n = 28). Statistical analyses were conducted using SAS software (version 9, SAS Institute). All statistical tests were two-sided.

Results

Characteristics of our study population according to a personal history of NMSC in mid-point of the follow-up (1998) are shown in Table 1. Participants with a history of NMSC were more likely to be older and tended to burn and have more severe sunburns. Participants with history of NMSC diagnosis were more likely to have red or blonde hair and to reside in high UV-index states. Other characteristics were similar between the exposure group and the non-exposure group.

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Table 1. Characteristics according to personal history of non-melanoma skin cancer in 1998.

doi:10.1371/journal.pmed.1001433.t001

We followed the HPFS participants from 1986 to 2008, for a total of 833,496 person-years. During this period, 1,577 cases of SCC, 10,422 cases of BCC, and 10,590 primary cancer cases other than NMSC were recorded. The mean time for the development of a primary cancer after NMSC was 116±47 mo. We followed the NHS from 1984 to 2008 (2,116,178 person-years) during which 2,322 cases of SCC, 21,781 cases of BCC, and 18,857 primary cancer cases other than NMSC were recorded. The mean time for the development of a primary cancer after NMSC was 156±71 mo.

A personal history of NMSC was associated with a higher risk of other primary cancers in men (RR = 1.15; 95% CI 1.09–1.22, p<0.0001) and women (RR = 1.26; 95% CI 1.21–1.31,p<0.0001) (Table 2). The association attenuated slightly when melanoma was excluded from the outcome in the analysis, but remained significant in men (RR = 1.11; 95% CI 1.05–1.18, p= 0.0007) and in women (RR = 1.20; 95% CI 1.15–1.25, p<0.0001). Age-standardized AR was 176 in men and 182 in women per 100,000 person-years. The association remained significant after we excluded those diagnosed with other primary cancers within the first 4 y of NMSC diagnosis in men (RR = 1.15; 95% CI 1.05–1.25) and women (RR = 1.19; 95% CI 1.11–1.28). In men, the association was significant according to BCC diagnosis (RR = 1.17; 95% CI 1.10–1.24) but not SCC diagnosis (RR = 1.01; 95% CI 0.87–1.17). In women, the association was significant for both BCC (RR = 1.25; 95% CI 1.20–1.30) and SCC diagnosis (RR = 1.24; 95% CI 1.10–1.39). We compared people with personal history of SCC with people with personal history of BCC on their risk of developing subsequent cancer, and no significant differences were found (Table S1). In addition, we have compared SCC in situ group with invasive SCC group and the group of SCC or BCC; the results are shown in Table S2. Compared to those with history of invasive SCC or those with history of SCC or BCC, individuals with history of SCC in situ were less likely to develop subsequent cancers. Such risk reduction was significant among women.

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Table 2. Overall and stratified analysis of risks of total subsequent primary cancers according to personal history of non-melanoma skin cancer in men and women.

doi:10.1371/journal.pmed.1001433.t002

No substantial differences were found in the stratified analysis according to age, UV-index, or BMI. When stratified by smoking status, significant associations were found in never-smokers (RR = 1.19; 95% CI 1.08–1.31 in men, and RR = 1.28; 95% CI 1.20–1.38 in women) and past smokers (RR = 1.12; 95% CI 1.03–1.22 in men, and RR = 1.27; 95% CI 1.20–1.35 in women), but not in current smokers (RR = 1.05; 95% CI 0.75–1.46 in men, and RR = 1.10; 95% CI 0.97–1.23 in women). The p-value for interaction was 0.046 for men and women combined.

For individual cancer sites (Table 3), a history of NMSC was associated with an increased risk of prostate cancer in men (RR = 1.11; 95% CI 1.02–1.20, p = 0.01). The age-standardized AR was 137 per 100,000 person-years. The RR was similar for fatal prostate cancer (RR = 1.17; 95% CI 0.89–1.53). A history of NMSC was also associated with an increased risk of melanoma in men (RR = 1.99; 95% CI 1.63–2.43, p<0.0001). The age-standardized AR was 116 per 100,000 person-years. In women, a history of NMSC was associated with an increased risk of breast cancer (RR = 1.19; 95% CI 1.11–1.28, p<0.0001; AR = 87 per 100,000 person-years), lung cancer (RR = 1.32; 95% CI 1.14–1.52, p = 0.0002; AR = 22 per 100,000 person-years), leukemia (RR = 1.30; 95% CI 1.00–1.69, p = 0.05; AR = 7 per 100,000 person-years), kidney cancer (RR = 1.48; 95% CI 1.10–1.99, p = 0.01; AR = 8 per 100,000 person-years), and melanoma (RR = 2.58; 95% CI 2.34 –2.98, p<0.0001; AR = 79 per 100,000 person-years). After taking into account the multiple comparisons for individual cancers (n = 28), the associations with breast cancer, lung cancer in women, and melanoma in both men and women remained significant. We analyzed SCC and BCC history separately for individual cancer sites. We observed different rates for second cancer development according to personal history of SCC and BCC. However, no statistically significant heterogeneity (p for heterogeneity ranged from 0.16 to 0.88) was found for any cancer site between BCC and SCC due to the limited power (Table S3).

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Table 3. Risks of subsequent primary cancers at different sites according to personal history of non-melanoma skin cancer in men and women.

doi:10.1371/journal.pmed.1001433.t003

Discussion

To the best of our knowledge, this is the largest prospective study on this topic. In this study, a total of 36,102 cases of NMSC and 29,447 cases of cancers other than NMSC were documented. Among those with a personal history of NMSC, we found a 15% increased risk in men and a 26% increased risk in women of developing a second primary cancer, compared with those who had no such history. A systematic review summarizing previous studies revealed that NMSC is associated with more than 10% increased risk of subsequent primary cancer in registry-based studies and nearly 50% increased risk in cohort studies [16]. Our study has extended previous findings by adding a prospective analysis in two large US cohorts with more than two decades of follow-up. The unique aspects of our study included stratified analyses by other risk factors, disentanglement of surveillance bias, and comprehensive adjustment for potential confounders.

It was speculated that the association between NMSC and subsequent cancer risk may be different among people living in locations with different UV-indexes. Specifically, southern regions have solar UV-B radiation levels that provide sufficient vitamin D to reduce the risk of cancer incidence, and thus inverse associations were more likely to be found. On the contrary, studies that found positive associations were mostly conducted in northern regions where UV-B radiation levels do not provide sufficient vitamin D [17],[18]. In our analysis stratified by UV-index, no substantial differences were found for the associations between NMSC and cancer risk among locations with different UV-indexes. To the extent that some cancers have been suggested to be reduced with higher vitamin D [19], it might be worth noting the fact that while colorectal cancer was not increased in this study, it was not decreased as would be expected if NMSC were a marker for sunlight and thus vitamin D exposure.

Intensified medical surveillance of persons with a history of NMSC is unlikely to explain the increased cancer incidence observed in our study. In our analysis, we adjusted for physical examination in the last 2 y, and the result changed little. After we excluded those diagnosed with other primary cancers within the first 4 y of NMSC diagnosis, the association remained significant. Compared to those with history of invasive SCC or with history of SCC or BCC, individuals with history of SCC in situ were less likely to develop subsequent cancers. These results suggested that the patients who carried NMSC precursors but did not develop skin cancers might be either less genetically susceptible or have lower exposure. In addition, for prostate cancer, the association remained significant for fatal prostate cancer. Furthermore, several cancers that we observed associations with are not ones that would be detected on routine screening. Moreover, at least one study that assessed deaths rather than cancer incidence also found increased cancer mortality in people with history of NMSC [20].

Several studies have observed an increased risk of NMSC after other cancers. In one study, chronic lymphocytic leukemia patients had an increased risk of death due to NMSC (RR = 17.0, 95% CI 14.4–19.8) [21]. In another study, among 14 different sites for first primary malignancies, 11 of these sites including prostate, breast, and leukemia were followed by an increased risk of skin cancer (for SCC, RR of 14.1 for males and 14.6 for females) [22]. However, while treatment of these primary cancers may predispose to subsequent skin cancers, most of the NMSC cases are cured by surgical excision without any systemic chemotherapy, and radiation, and their concomitant side effects, including possible carcinogenicity. In addition, the similarity between the age-adjusted and multivariate-adjusted RRs demonstrated that the observed association between NMSC and subsequent cancers is unlikely to be explained by confounding from smoking, obesity, vitamin use, exercise, or any of the other measured risk factors that we controlled for.

The link between NMSC and risk of other cancers is likely to represent an etiologic association. For melanoma especially, the link may be due to sun exposure. For other cancers, while there are several explanations [23][28] of the association between NMSC and the risk of subsequent cancer, studies have found that certain genetic markers underlying skin cancer are also associated with other cancer types [29]. It is biologically plausible that deficiencies of pathways responsible for protecting against cellular transformation in multiple tissues, such as DNA repair or immune responses, may act systemically and play a role in cutaneous and internal carcinogenesis.

Humans have evolved several DNA repair pathways dealing with damage [30]. The nucleotide excision repair (NER) pathway is responsible for the repair of a wide variety of DNA damage that leads to distortion of the DNA helix. Such bulky DNA adducts include UV-induced photoproducts, smoking-related benzo(a)pyrene diolepoxide (BPDE)-DNA adducts, and other DNA damage induced by chemical carcinogens. Reduced capacity of the NER has been shown to confer susceptibility to certain cancers in the general population, including melanoma, BCC, SCC, SCC of head and neck, lung cancer, breast cancer, and bladder cancer[31][34]. Personal history of NMSC may be a marker of susceptibility due to reduced DNA repair capacity and it may predict the risk of subsequent cancer development.

The NER activity has been shown to be tissue-specific. For example, relatively low NER efficiency was observed in oral tissues [35]. Both rapidly proliferating tissues (e.g., kidney) and slowly proliferating tissues (e.g., lung) exhibit higher demand for NER capacity upon stimulation to proliferation [36]. The DNA repair system consists of several distinct pathways with many subcomponents, each interacting and overlapping with one another in order to achieve genomic stability and high fidelity. Some tissues, such as breast, lack redundant systems of DNA repair that are present in other tissues [37],[38]. Defects in DNA repair would be expected to have greater impact in such tissues without extensive DNA repair redundancy. In addition, a number of studies have suggested a role of sex hormone (e.g., estrogen and androgen) in the regulation of DNA repair activity in breast and prostate cancers [39]. After correction for multiple comparisons for individual types of cancers, the significant association remained for breast cancer, lung cancer in women, and melanoma in both men and women. Even though the positive associations are biologically plausible, we cannot rule out the possibility of chance findings for each individual cancer site.

In our analysis stratified by smoking status, significant association was found among never and past smokers. Because the NER enzymes recognize bulky DNA adducts including both UV-induced photoproducts and smoking-related BPDE-DNA adducts, the interaction between smoking status and history of NMSC highlights the potential role of the NER pathway in the development of second cancers. However, the effect of inherited insufficient capacity of the NER indicated by history of NMSC is only apparent among non-current smokers and for lung cancer in women. Further mechanistic investigation is warranted.

Sub-optimal immune surveillance could be another common susceptibility factor for both cutaneous and internal cancers. Malignant progression is accompanied by profound immune suppression that interferes with an effective antitumor response and tumor elimination [40]. Impaired immunity has been implicated as a non-site-specific determinant of cancer risk [41]. In addition, UV radiation can also cause immunosuppression. UV exposure adversely affects the skin immune system by diminishing antigen-presenting cell function, inducing immunosuppressive cytokine production, and modulating contact and delayed-type hypersensitivity reactions [42],[43], all of which can reduce the body’s surveillance for tumor cells [44],[45]. UV suppresses immune reactions locally, but can also affect the immune system in a systemic fashion when higher UV doses are given [46],[47]. UV radiation affects immune surveillance by modulating the balance between an effective immune response and immune tolerance of an emerging cancer [41]. We did not observe an association between UV-index and the risk of cancer except for skin cancer in our study, which makes this explanation less likely for our findings.

The identification of BCC cases in this study was based on self-report without pathological confirmation. However, the participants in the two cohorts were nurses and health professionals. The validity of their reports was expected to be high, and it has been proven in our validation studies [13],[14]. In addition, previous studies of BCC in the NHS using the self-reported cases identified both constitutional and sun exposure risk factors as expected, such as lighter pigmentation, less childhood and adolescent tanning tendency, higher tendency to sunburn, and tanning salon attendance [48],[49]. We recently confirmed the MC1R gene as the top BCC risk locus using the NHS and HPFS samples [50]. These data together suggest that the bias due to self-report of BCC is likely to be minimal in our study. Moreover, the potential under-report of BCC diagnosis would be expected to bias observed associations toward the null, and such bias would not explain the positive associations that we found.

The strengths of our study included the prospective cohort design and updated assessment of cancer diagnosis and other risk factors every 2 y, more than two decades of follow-up, and a large number of incident skin cancer cases. We had detailed data on related covariates for stratified analyses and comprehensive adjustment for potential confounders. All the participants were health professionals, minimizing potential confounding by educational attainment or differential access to health care. Nevertheless, we cannot completely exclude residual confounding, and our findings may not assign causality. Although the observed significant associations in breast cancer, lung cancer in women, and melanoma in both men and women remained significant after correction for multiple comparisons, we cannot absolutely rule out chance findings for individual cancer sites, and the underlying mechanism for the associations found in specific cancer sites is not entirely clear. In addition, the significant associations for some individual cancers did not meet the adjusted p-value threshold because of their limited sample size.

We cannot estimate the recurrence rate of NMSC or subsequent cancer risk among people with multiple NMSC because we only recorded the first report of each type of skin cancer in both cohorts. We do not have data for non-whites in this study, and our results cannot be generalized to non-whites owing to the dramatic difference in skin cancer incidence among different races. In summary, we observed a modestly increased risk of other cancers among individuals with a history of NMSC. Because our study was observational, these results should be interpreted cautiously and are insufficient evidence to alter current clinical recommendations. Nevertheless, these data support a need for continued investigation of the potential mechanisms underlying this relationship.

SOURCE:

http://www.plosmedicine.org/article/info%3Adoi%2F10.1371%2Fjournal.pmed.1001433;jsessionid=55BB9D9B87F79FF1CA7594C56F407F14

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Reporter: Aviva Lev-Ari, PhD, RN

 

 

Changes in breast density point to tamoxifen‘s effectiveness

By Kate Madden Yee, AuntMinnie.com staff writer

April 22, 2013 — If women being treated with tamoxifen for breast cancer see their breast density drop, they may have a 50% lower risk of dying from the disease, according to a new study by Swedish researchers published online April 22 in the Journal of Clinical Oncology.

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In a study that hints at a role for breast density measurement in predicting therapy response, researchers at Karolinska Institutet in Stockholm found that women who had a relative reduction of more than 20% in the absolute dense area of their breast tissue during the course of tamoxifen treatment had a 50% reduction in breast cancer mortality over a span of 15 years, compared with women who had little or no change (JCO, April 22, 2013).

Tamoxifen is usually given over five years to prevent the recurrence of breast cancer in women who have completed their primary treatment. The researchers conducted the study to assess whether there was a link between reduced tissue density and the effectiveness of tamoxifen therapy.

Dr. Per Hall

Dr. Per Hall from Karolinska Institutet.

No method has been available for assessing which women are likely to respond to tamoxifen and not relapse with breast cancer, according to Dr. Per Hall and colleagues.

“To the best of our knowledge, this is the first time mammographic density change has been used as a prognostic marker of response to tamoxifen,” Hall and colleagues wrote. “We observed that women treated with tamoxifen who experienced mammographic density reduction were associated with substantially better long-term breast-cancer-specific survival. If validated, mammographic density change has the potential to be an early marker for therapy response.”

For the study, Hall and colleagues included data collected in Sweden between 1993 and 1995 from 974 postmenopausal patients with breast cancer who had both baseline and follow-up mammograms. Of these, 474 patients received tamoxifen treatment and 500 did not. The team measured mammographic density using a statistical method that expressed the data as absolute dense area.

During the follow-up period, 121 patients (12.4%) died from breast cancer. But women who were treated with tamoxifen and who experienced a relative density reduction of more than 20% had a 50% lower risk of death, compared with women whose breast density didn’t change, the team found.

In the group that was not treated with tamoxifen, there was no statistically significant association between mammographic density change and survival, and the survival advantage was not found when absolute dense areas at baseline or follow-up were evaluated separately.

The findings come on the heels of a recommendation issued on April 15 from the U.S. Preventive Services Task Force (USPSTF) regarding the use of tamoxifen and a related drug, raloxifene, as preventive measures against breast cancer in asymptomatic women. The USPSTF suggests that women who are at increased risk for breast cancer talk to their physician about the potential benefits and harms of the drugs to prevent breast cancer; women who are not at increased risk should not use them.

If further research confirms the Swedish group’s findings, mammographic density change has the potential to be an early marker for therapy response, Hall and colleagues wrote. In fact, given ongoing developments in automatic algorithms for mammographic density measurement, using density change to monitor the effectiveness of treatment could be a cost-effective clinical tool.

“What’s needed is accurate measurement of mammographic density,” Hall said in a statement released by the Karolinska Institutet. “Measuring changes in density can be a simple and cheap means of assessing the effect of the treatment. If a patient is not responding to tamoxifen, maybe they should be given a different drug.”

Related Reading

ARRS: Breast US spots missed cancers in dense breasts, April 18, 2013

AHRA backs Are You Dense Advocacy, April 10, 2013

Calif. breast density bill goes into effect, April 1, 2013

Rads judge breast density the same for digital, analog mammo, February 28, 2013

Yearly screening breast US benefits women with dense tissue, December 4, 2012
Copyright © 2013 AuntMinnie.com

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Whole exome somatic mutations analysis of malignant melanoma contributes to the development of personalized cancer therapy for this disease

Author: Ziv Raviv, PhD

Introduction

Cutaneous melanoma is a type of skin cancer that originates in melanocytes, the cells that are producing melanin. While being the least common type of skin cancer, melanoma is the most aggressive one with invasive characteristics and accounts for the majority of death incidences among skin cancers. Melanoma has an annual rate of 160,000 new cases and 48,000 deaths worldwide. Melanoma affects mainly Caucasians exposed to sun high UV irradiation. Among the genetic factors that characterize the disease, BRAF mutation (V600E) is found in most cases of melanoma (80%).  Awareness toward risk factors of melanoma should lead to prevention and early detection*. There are several developmental stages (I-IV) of the disease, starting from local non-invasive melanoma, through invasive and high risk melanoma, up to metastatic melanoma. As with other cancers, the earlier stage melanoma is being detected, the better odds for full recovery are. Treatment is usually involving surgery to remove the local tumor and its margins, and when necessary also to remove the proximal lymph node(s) that drain the tumor. In high stages melanoma, adjuvant therapy is given in the form of chemotherapy (Dacarbazine and Temozolomide) and immunotherapy (IL-2 and IFN). Until recently no useful treatment was available for metastatic melanoma. However, research efforts had led to the development of two new drugs against metastatic melanoma: Vemurafenib (Zelboraf), a B-Raf inhibitor; and Ipilimumab (Yervoy), a monoclonal antibody that blocks the inhibitory signal of cytotoxic T lymphocyte-associated antigen 4 (CTLA-4). Both drugs are now available for clinical use presenting good results.

Personalized therapy for melanoma

In an attempt to develop personalized therapies for malignant melanoma, a unique strategy has been taken by the group of Prof. Yardena Samuels at the NIH (now situated at the WIS) to identify recurring genetic alterations of metastatic cutaneous melanoma. The researchers approach employed the collections of hundreds of tumors samples taken from metastasized melanoma patients together with matched normal blood tissues samples. The samples are undergoing exome sequencing for the analysis of somatic mutations (namely mutations that evolved during the progress of the disease to the stage of metastatic melanoma, unlike genomic mutations that may have contribute to the formation of the disease). The discrimination of such tumor related somatic mutations is done by comparison to the exome sequencing of the patient’s matched blood cells DNA. In addition, the malignant cells derived from the removed cancer tissue of each patient are extracted to form a cell line and are grown in culture. These cells are easily cultivate in culture with no special media supplements, nor further genetic manipulations such as hTERT are needed, and are extremely aggressive as determined by various cell culture and in vivo tests. The ability to grow these primary tumor-derived cell lines in culture has a great value as a tool for studying and characterizing the biochemical, functional, and clinical aspects of the mutated genes identified.

In one study [1] Samuels and her colleagues performed this sequencing process for mutation analysis for the protein tyrosine kinase (PTK) gene family, as PTKs are frequently mutated in cancer. Using high-throughput gene sequencing to analyze the entire PTK gene family, the researchers have identified 30 somatic mutations affecting the kinase domains of 19 PTKs and subsequently evaluated the entire coding regions of the genes encoding these 19 PTKs for somatic mutations in 79 melanoma samples. The most frequent mutations were found in ERBB4, a member of the EGFR/ErbB family of receptor tyrosine kinase (RTK), were 19% of melanoma patients had such mutations. Seven missense mutations in the ERBB4 gene were found to induce increased kinase activity and transformation capability. Melanoma derived cell lines that were expressing these mutant ERBB4 forms had reduced cell growth after silencing ERBB4 by RNAi or after treatment with the ERBB inhibitor Lapatinib. Lapatinib is already in use in the clinic for the treatment of HER2 (ErbB2) positive breast cancers patients. Following this study, a clinical trial is now conducted with this drug to evaluate its effect in cutaneous metastatic melanoma patients harboring mutations in ERBB4.

In another study of this group [2], the scientists employed the exome sequencing method to analyze the somatic mutations of 734 G protein coupled receptors (GPCRs) in melanoma. GPCRs are regulating various signaling pathways including those that affect cell growth and play also important role in human diseases. This screen revealed that GRM3 gene that encode the metabotropic glutamate receptor 3 (mGluR3), was frequently mutated and that one of its mutations clustered within one position. Mutant GRM3 was found to selectively regulate the phosphorylation of MEK1 leading to increased anchorage-independent cell growth and cellular migration. Tumor derived melanoma cells expressing mutant GRM3 exhibited reduced cell growth and migration upon knockdown of GRM3 by RNAi or by treatment with the selective MEK inhibitor, Selumetinib (AZD-6244), a drug that is being testing in clinical trials. Altogether, the results of this study point to the increased violent characteristics of melanomas bearing mutational GRM3.

In a third study, melanoma samples were examined for somatic mutations in 19 human genes that encode ADAMTS proteins [3]. Some of the ADAMTS genes have been suggested before to have implication in tumorigenesis. ADAMTS18, which was previously found to be a candidate cancer gene, was found in this study to be highly mutated in melanoma. ADAMTS18 mutations were biologically examined and were found to induce an increased proliferation of melanoma cells, as well as increased cell migration and metastasis. Moreover, melanoma cells expressing these mutated ADAMTS18 had reduced cell migration after RNAi-mediated knockdown of ADAMTS18. Thus, these results suggest that genetic alteration of ADAMTS18 plays a major role in melanoma tumorigenesis. Since ADAMTS genes encode extracellular proteins, their accessibility to systematically delivered drugs makes them excellent therapeutic targets.

Conclusive remarks

The above illustrated research approach intends to discover frequent melanoma-specific mutations by employing high-throughput whole exome and genome sequencing means. For the most highly mutated genes identified, the biochemical, functional, and clinical aspects are being characterized to examine their relevancy to the disease outcomes. This approach therefore introduces new opportunities for clinical intervention for the treatment of cutaneous melanoma. In addition to the discovery of novel highly mutated genes, this approach may also help determine which pathways are altered in melanoma and how these genes and pathways interact. Finding melanoma-associated highly mutated genes could lead to personalized therapeutics specifically targeting these altered genes in individual melanomas. Along with the opportunity to develop new agents to treat melanoma, the approach takes advantage of existing anti-cancer drugs, utilizing them to treat these mutated genes melanoma individuals. In addition to their potential for therapeutics, the discovery of highly mutated genes in melanoma patients may lead to the discovery of new markers that may assist the diagnosis of the disease. The implications of these screenings findings on other types of cancer bearing common pathways similar to melanoma should be examined as well. Finally, this elegant approach should be adopted in research efforts of other cancer types.

* Special review will be published further in the cancer prevention section of Pharmaceutical Intelligence

References

1. Prickett TD, Agrawal NS, Wei X, Yates KE, Lin JC, Wunderlich JR, Cronin JC, Cruz P, Rosenberg SA, Samuels Y (2009) Analysis of the tyrosine kinome in melanoma reveals recurrent mutations in ERBB4. Nat Genet 41 (10):1127-1132

2. Prickett TD, Wei X, Cardenas-Navia I, Teer JK, Lin JC, Walia V, Gartner J, Jiang J, Cherukuri PF, Molinolo A, Davies MA, Gershenwald JE, Stemke-Hale K, Rosenberg SA, Margulies EH, Samuels Y (2011) Exon capture analysis of G protein-coupled receptors identifies activating mutations in GRM3 in melanoma. Nat Genet 43 (11):1119-1126

3. Wei X, Prickett TD, Viloria CG, Molinolo A, Lin JC, Cardenas-Navia I, Cruz P, Rosenberg SA, Davies MA, Gershenwald JE, Lopez-Otin C, Samuels Y (2010) Mutational and functional analysis reveals ADAMTS18 metalloproteinase as a novel driver in melanoma. Mol Cancer Res 8 (11):1513-1525

Related articles on melanoma on this open access online scientific journal:

1.  In focus: Melanoma Genetics. Curator: Ritu Saxena, Ph.D.

2.  In focus: Melanoma therapeutics. Author and Curator: Ritu Saxena, Ph.D.

3.  A New Therapy for Melanoma.  Reporter- Larry H Bernstein, M.D.

4. Thymosin alpha1 and melanoma. Author, Editor: Tilda Barliya, Ph.D.

5. Exome sequencing of serous endometrial tumors shows recurrent somatic mutations in chromatin-remodeling and ubiquitin ligase complex genes. Reporter and Curator: Dr. Sudipta Saha, Ph.D.

6. How Genome Sequencing is Revolutionizing Clinical Diagnostics. Reporter: Aviva Lev-Ari, PhD, RN.

7. Issues in Personalized Medicine in Cancer: Intratumor Heterogeneity and Branched Evolution Revealed by Multiregion Sequencing. Curator and Reporter: Stephen J. Williams, Ph.D.

 

 

 

 

 

 

 

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Reporter: Aviva Lev-Ari, PhD, RN

Dr. Lev-Ari met  Emory Cardiologist, Arshed Quyyumi following his talk as Guest Speaker at Tufts, Cardiology Colloquium, Tufts Medical School, Boston in 8/2007. They corresponded about CVD and Stem Cell therapy post MI.

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Emory Cardiologists Arshed Quyyumi And Javed Butler Join Stemedica Cardiology Division Scientific Advisory Board

Stemedica Cell Technologies, Inc., a leader in adult allogeneic stem cell manufacturing, research and development, announced today that Professors Arshed Quyyumi, MD, FRCP, FACC and Javed Butler, MD, MPH, FACC, FAHA of the Emory Clinical Cardiovascular Research Institute have agreed to join the Scientific Advisory Board for Stemedica’s Cardiology Division.

San Diego, CA (PRWEB) April 22, 2013

Stemedica Cell Technologies, Inc., a leader in adult allogeneic stem cell manufacturing, research and development, announced today that Professors Arshed Quyyumi, MD, FRCP, FACC and Javed Butler, MD, MPH, FACC, FAHA of the Emory Clinical Cardiovascular Research Institute have agreed to join the Scientific Advisory Board for Stemedica’s Cardiology Division.

Dr. Arshed A. Quyyumi has been involved in clinical translational research in cardiovascular diseases for over 25 years. His research focuses on vascular biology, angiogenesis, progenitor cell biology, mechanisms of myocardial ischemia, and the role of genetic and environmental risks on vascular disease. In 2001 he was appointed Professor of Medicine in the Division of Cardiology at the Emory University School of Medicine. In 2010, he was named Co-Director of the Emory Clinical Cardiovascular Research Institute (ECCRI). Dr. Quyyumi serves on the editorial boards of several national journals and is a reviewer for the National Institute of Health’s (NIH) National Heart, Lung and Blood Institute Study Sections. Dr. Quyyumi has authored more than 220 peer-reviewed publications and has been an invited speaker and session chair at many scientific meetings and conferences. During his academic career, Dr. Quyyumi has managed more than 50 NIH, industry-funded, or investigator-initiated projects, including numerous clinical trials.

Dr. Javed Butler’s research focuses on the disease progression, outcomes, and prognosis determination in patients with heart failure, with special emphasis on patients undergoing cardiac transplantation and left ventricular assist device placement. Before moving to Emory University where he is a professor of cardiology, he was Director for the Heart and Heart-Lung Transplant programs at Vanderbilt University. He also serves as the Deputy Chief Science Advisor for the American Heart Association. Additionally, he serves various editorial responsibilities for the Journal of the American College of Cardiology, Journal of Cardiac Failure, American Heart Journal, Journal of the American College of Cardiology – Heart Failure, Heart Failure Clinics, Current Heart Failure Report, and Congestive Heart Failure. He is board certified in Cardiology, Internal Medicine, Advanced Heart Failure and Transplantation Medicine, and Nuclear Cardiology. Dr. Butler also serves on the Executive Council of the Heart Failure Society of America. He has published over 175 peer reviewed papers and has participated in over 50 federally funded and non-federally funded clinical trials. He has served on several steering committees, events committees and data safety monitoring committees for multi-center clinical trials.

Stephen Epstein, M.D., Chairman of the Medical & Scientific Advisory Board for Stemedica’s Cardiology Division, commented, “I am delighted to welcome Drs. Quyyumi and Butler to the cardiology team. I believe our collective breadth of experience will provide Stemedica with both perspective and direction as the company expands its efforts in cardiovascular medicine.”

Stemedica recently formed its Cardiology Division due to the considerable progress in the translational application of its ischemia tolerant adult allogeneic stem cells for cardiovascular diseases. The Company gained FDA approval for a multi-center Phase II clinical trial for intravenous treatment of AMI with its Stemedyne™ MSC product. Regulatory approval has also been granted to the National Medical Research Center in Astana, Kazakhstan for a Phase III trial with Stemedyne™ MSC. A Phase II clinical trial for chronic heart failure has begun at Hospital Angeles in Mexico. The Cardiology Division Scientific Advisory Board was organized to support these and future efforts.

Sergey Sikora, Ph.D., M.D., President of Stemedica’s Cardiovascular Division noted, “Emory has been one of the leaders in cardiology and we are honored to have physicians of Drs. Quyyumi and Butler’s statures participate with us in cardiovascular translational medicine.”

Nikolai Tankovich, MD, PhD, FASLMS, President and Chief Medical Officer of Stemedica commented, “We are pleased by the impressive and promising outcomes that Stemedica’s adult stem cells are experiencing in cardiology. We view this area as critical for the success of Stemedica and have worked diligently to get the best experts to advise us in this field.”

About Stemedica Cell Technologies, Inc. http://www.stemedica.com
Stemedica Cell Technologies, Inc. is a specialty bio-pharmaceutical company that is committed to the manufacturing and development of best-in-class allogeneic adult stem cells and stem cell factors for use by approved research institutions and hospitals for pre-clinical and clinical (human) trials. The company is a government licensed manufacturer of clinical grade stem cells and is approved by the FDA for clinical trials in ischemic stroke, cutaneous photoaging and myocardial infarct. Stemedica is currently developing regulatory pathways for a number of other medical indications using adult allogeneic stem cells. The company is headquartered in San Diego, California.

For more information regarding Stemedica Cell Technologies, Inc., contact Dave McGuigan at dmcguigan (at) stemedica.com.

http://www.prweb.com/releases/stemedica-stem-cells/cardiology-Quyyumi-Butler/prweb10655555.htm

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