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Archive for the ‘Pharmaceutical Drug Discovery’ Category


Live Conference Coverage @Medcitynews Converge 2018 @Philadelphia: Promising Drugs and Breaking Down Silos

Reporter: Stephen J. Williams, PhD

Promising Drugs, Pricing and Access

The drug pricing debate rages on. What are the solutions to continuing to foster research and innovation, while ensuring access and affordability for patients? Can biosimilars and generics be able to expand market access in the U.S.?

Moderator: Bunny Ellerin, Director, Healthcare and Pharmaceutical Management Program, Columbia Business School
Speakers:
Patrick Davish, AVP, Global & US Pricing/Market Access, Merck
Robert Dubois M.D., Chief Science Officer and Executive Vice President, National Pharmaceutical Council
Gary Kurzman, M.D., Senior Vice President and Managing Director, Healthcare, Safeguard Scientifics
Steven Lucio, Associate Vice President, Pharmacy Services, Vizient

What is working and what needs to change in pricing models?

Robert:  He sees so many players in the onStevencology space discovering new drugs and other drugs are going generic (that is what is working).  However are we spending too much on cancer care relative to other diseases (their initiative Going Beyond the Surface)

Steven:  the advent of biosimilars is good for the industry

Patrick:  large effort in oncology, maybe too much (750 trials on Keytruda) and he says pharma is spending on R&D (however clinical trials take large chunk of this money)

Robert: cancer has gotten a free ride but cost per year relative to benefit looks different than other diseases.  Are we overinvesting in cancer or is that a societal decision

Gary:  maybe as we become more specific with precision medicines high prices may be a result of our success in specifically targeting a mutation.  We need to understand the targeted drugs and outcomes.

Patrick: “Cancer is the last big frontier” but he says prices will come down in most cases.  He gives the example of Hep C treatment… the previous only therapeutic option was a very toxic yearlong treatment but the newer drugs may be more cost effective and safer

Steven: Our blockbuster drugs could diffuse the expense but now with precision we can’t diffuse the expense over a large number of patients

President’s Cancer Panel Recommendation

Six recommendations

  1. promoting value based pricing
  2. enabling communications of cost
  3. financial toxicity
  4. stimulate competition biosimilars
  5. value based care
  6. invest in biomedical research

Patrick: the government pricing regime is hurting.  Alot of practical barriers but Merck has over 200 studies on cost basis

Robert:  many concerns/impetus started in Europe on pricing as they are a set price model (EU won’t pay more than x for a drug). US is moving more to outcomes pricing. For every one health outcome study three studies did not show a benefit.  With cancer it is tricky to establish specific health outcomes.  Also Medicare gets best price status so needs to be a safe harbor for payers and biggest constraint is regulatory issues.

Steven: They all want value based pricing but we don’t have that yet and there is a challenge to understand the nuances of new therapies.  Hard to align all the stakeholders together so until some legislation starts to change the reimbursement-clinic-patient-pharma obstacles.  Possibly the big data efforts discussed here may help align each stakeholders goals.

Gary: What is the data necessary to understand what is happening to patients and until we have that information it still will be complicated to determine where investors in health care stand at in this discussion

Robert: on an ICER methods advisory board: 1) great concern of costs how do we determine fair value of drug 2) ICER is only game in town, other orgs only give recommendations 3) ICER evaluates long term value (cost per quality year of life), budget impact (will people go bankrupt)

4) ICER getting traction in the public eye and advocates 5) the problem is ICER not ready for prime time as evidence keeps changing or are they keeping the societal factors in mind and they don’t have total transparancy in their methodology

Steven: We need more transparency into all the costs associated with the drug and therapy and value-based outcome.  Right now price is more of a black box.

Moderator: pointed to a recent study which showed that outpatient costs are going down while hospital based care cost is going rapidly up (cost of site of care) so we need to figure out how to get people into lower cost setting

Breaking Down Silos in Research

“Silo” is healthcare’s four-letter word. How are researchers, life science companies and others sharing information that can benefit patients more quickly? Hear from experts at institutions that are striving to tear down the walls that prevent data from flowing.

Moderator: Vini Jolly, Executive Director, Woodside Capital Partners
Speakers:
Ardy Arianpour, CEO & Co-Founder, Seqster @seqster
Lauren Becnel, Ph.D., Real World Data Lead for Oncology, Pfizer
Rakesh Mathew, Innovation, Research, & Development Lead, HealthShareExchange
David Nace M.D., Chief Medical Officer, Innovaccer

Seqster: Seqster is a secure platform that helps you and your family manage medical records, DNA, fitness, and nutrition data—all in one place. Founder has a genomic sequencing background but realized sequence  information needs to be linked with medical records.

HealthShareExchange.org :

HealthShare Exchange envisions a trusted community of healthcare stakeholders collaborating to deliver better care to consumers in the greater Philadelphia region. HealthShare Exchange will provide secure access to health information to enable preventive and cost-effective care; improve quality of patient care; and facilitate care transitions. They have partnered with multiple players in healthcare field and have data on over 7 million patients.

Innovacer

Data can be overwhelming, but it doesn’t have to be this way. To drive healthcare efficiency, we designed a modular suite of products for a smooth transition into a data-driven world within 4 weeks. Why does it take so much money to move data around and so slowly?

What is interoperatibility?

Ardy: We knew in genomics field how to build algorithms to analyze big data but how do we expand this from a consumer standpoint and see and share your data.

Lauren: how can we use the data between patients, doctors, researchers?  On the research side genomics represent only 2% of data.  Silos are one issue but figuring out the standards for data (collection, curation, analysis) is not set. Still need to improve semantic interoperability. For example Flatiron had good annotated data on male metastatic breast cancer.

David: Technical interopatabliltiy (platform), semantic interopatability (meaning or word usage), format (syntactic) interopatibility (data structure).  There is technical interoperatiblity between health system but some semantic but formats are all different (pharmacies use different systems and write different prescriptions using different suppliers).  In any value based contract this problem is a big issue now (we are going to pay you based on the quality of your performance then there is big need to coordinate across platforms).  We can solve it by bringing data in real time in one place and use mapping to integrate the format (need quality control) then need to make the data democratized among players.

Rakesh:  Patients data should follow the patient. Of Philadelphia’s 12 health systems we had a challenge to make data interoperatable among them so tdhey said to providers don’t use portals and made sure hospitals were sending standardized data. Health care data is complex.

David: 80% of clinical data is noise. For example most eMedical Records are text. Another problem is defining a patient identifier which US does not believe in.

 

 

 

 

Please follow on Twitter using the following #hash tags and @pharma_BI

#MCConverge

#cancertreatment

#healthIT

#innovation

#precisionmedicine

#healthcaremodels

#personalizedmedicine

#healthcaredata

And at the following handles:

@pharma_BI

@medcitynews

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In 2018 Cardiovascular PharmacoTherapy Market: Anti-thrombotic Drug Class Segment will continue to bring in the biggest profit and dominate production

Reporter: Aviva Lev-Ari, PhD, RN

Who were the top players in cardiovascular disease in 2017?

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CHI’s Discovery on Target, Sheraton Boston, Sept. 25-28, 2018

Reporter: Aviva Lev-Ari, PhD, RN

 

ANNOUNCEMENT

Leaders in Pharmaceutical Business Intelligence (LPBI) Group is a selected CHI Business Partner for Media Communication for this event as well a provider of REAL TIME PRESS COVERAGE for this cardinal event in the domain of  Drug Discovery and Drug Delivery.

Dr. Aviva Lev-Ari, PhD, RN, Editor-in-Chief, PharmaceuticalIntelligence.com  will be in attendance covering this event for the Press using Social Media via 12 Channels

LOGO of LPBI Group

Follow us on ALL our Media Communication Channels:

Channels for e-Marketing of Biotech Conferences

  • Our Journal has 1,373,977  eReaders on 1/29/2018, for All Time and 7,283 Scientific Comments

http://pharmaceuticalintelligence.com

  • Aviva’s – +6,430 BIOTECH Followers on LinkedIn

http://www.linkedin.com/in/avivalevari

  • Aviva is a Member of +60 LinkedIn Groups in Biotech related fields

https://www.linkedin.com/groups/my-groups

  • LPBI Group’s FaceBook Page

http://www.facebook.com/LeadersInPharmaceuticalBusinessIntelligence

  • LPBI Group’s Twitter Account

http://twitter.com/pharma_BI

  • LPBI Group’s Company’s Page on LinkedIn

https://www.linkedin.com/company/9325543?trk=tyah&trkInfo=clickedVertical%3Acompany%2CclickedEntityId%3A9325543%2Cidx%3A1-1-1%2CtarId%3A1439226813927%2Ctas%3ALeaders%20in%20Pharmaceutica

 

 

For UPDATES on this Cardinal Conference and for REGISTRATION, go to 

http://www.discoveryontarget.com/?utm_source=partner

 

For PROGRAMS, go to 

http://www.discoveryontarget.com/programs

What is the Role of the Editor-in-Chief at PharmaceuticalIntelligence.com 

Editor-in-Chief’s Roles and Accomplishments

1        Curation Methodology Development

Leadership we provide on curation of scientific findings in the eScientific publishing for Medical Education contents.

In Section 1, the Leadership we provide on curation of scientific findings in the eScientific publishing for Medical Education contents is demonstrated by a subset of several outstanding curations with high electronic Viewer volume. Each article included presents unique content contribution to Medical Clinical Education.

·       These articles are extracted from the list of all Journal articles with >1,000 eReaders, 4/28/2012 to 1/29/2018.

Article Title,         # of electronic Viewers,         Author(s) Name

Is the Warburg Effect the Cause or the Effect of Cancer: A 21st Century View?                      16,114 Larry H. Bernstein, MD, FCAP

Do Novel Anticoagulants Affect the PT/INR? The Cases of XARELTO (rivaroxaban) and PRADAXA (dabigatran) 11,606 Vivek Lal, MBBS, MD, FCIR,

Justin D. Pearlman, MD, PhD, FACC and

Aviva Lev-Ari, PhD, RN

Clinical Indications for Use of Inhaled Nitric Oxide (iNO) in the Adult Patient Market: Clinical Outcomes after Use, Therapy Demand and Cost of Care

 

 5,865 Aviva Lev-Ari, PhD, RN
Peroxisome proliferator-activated receptor (PPAR-gamma) Receptors Activation: PPARγ transrepression for Angiogenesis in Cardiovascular Disease and PPARγ transactivation for Treatment of Diabetes                  1,919 Aviva Lev-Ari, PhD, RN  

 

Bystolic’s generic Nebivolol – Positive Effect on circulating Endothelial Progenitor Cells Endogenous Augmentation  1,059 Aviva Lev-Ari, PhD, RN

 

Triple Antihypertensive Combination Therapy Significantly Lowers Blood Pressure in Hard-to-Treat Patients with Hypertension and Diabetes  1,339 Aviva Lev-Ari, PhD, RN

 

Clinical Trials Results for Endothelin System: Pathophysiological role in Chronic Heart Failure, Acute Coronary Syndromes and MI – Marker of Disease Severity or Genetic Determination?  1,472 Aviva Lev-Ari, PhD, RN
Treatment of Refractory Hypertension via Percutaneous Renal Denervation  1,085 Aviva Lev-Ari, PhD, RN

2        Content Creation and Key Opinion Leader (KOL) Recognition

2.1     Volume of Articles in the Journal and in the 16 Volume-BioMed e-Series

Select

Aviva Lev-Ari, PhD, RN 2012pharmaceutical

3,064 Articles

·       All  (5,288)

avivalev-ari@alum.berkeley.edu Administrator 3064

2.1     Volume of Articles in the Journal and in the 16 Volume-BioMed e-Series

1.   Volume of Articles in the Journal since Journal inception on 4/28/2012:

·       Total articles by ALL authors in Journal Archive on 1/29/2018 = 5,288

·       ALL articles/posts Authored, Curated, Reported by Aviva Lev-Ari, PhD, RN = 3,064

2.   Volume of Articles in the 16 Volume-BioMed e-Series

·    Editorial & Publication of Articles in e-Books by Leaders in Pharmaceutical Business Intelligence: Contributions of Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2014/10/16/editorial-publication-of-articles-in-e-books-by-leaders-in-pharmaceutical-business-intelligence-contributions-of-aviva-lev-ari-phd-rn/

·       LPBI Group’s Founder: Biography and Bibliographies – Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/founder/

 

2.2     Digital Presence measured by eViews: Clicks on article by Author Name

Top Authors for all days ending 2018-01-29 (Summarized)

All Time

Author Name electronic Views
Aviva Lev-Ari, PhD, RN [2012pharmaceutical]

352,153

 

Our TEAM 5,934  

 

Founder 3,257
BioMed e-Series 3,140

 

Journal PharmaceuticalIntelligence.com 2,214
About 2,054
  VISION   2,803  

 


LPBI Group
            1,201

2.3     Digital KOL Parameters

Key Opinion Leader (KOL) – Aviva Lev-Ari, PhD, RN, as Evidenced by

https://pharmaceuticalintelligence.com/2016/07/21/key-opinion-leader-kol-aviva-lev-ari-phd-rn-as-evidenced-by/

 

3        Team building: Editors and Expert, Authors, Writers

Our Team

Selection of Journal’s Chief Scientific Officer (CSO) and BioMed e-Series Content Consultant (CC): Series B, C, D, E

L.H. Bernstein, MD, FCAP

Editorial & Publication of Articles in e-Books by  Leaders in Pharmaceutical Business Intelligence:  Contributions of Larry H Bernstein, MD, FCAP

https://pharmaceuticalintelligence.com/2014/10/16/editorial-publication-of-articles-in-e-books-by-leaders-in-pharmaceutical-business-intelligence-contributions-of-larry-h-bernstein-md-fcap/

4        Book Title Generation and Cover Page Design

As BioMed e-Series Editor–in-Chief, I was responsible for the following functions of product design and product launch

·       16 Title creations for e-Books

·       Designed 16 Cover Pages for a 16-Volume e-Books e-Series in BioMed

·       Designed Series A eTOCs and approved of all 16 electronic Table of Contents (eTOCs), working in tandem with all the Editors of each volume and all the Author contributors of article contents in the Journal.

·       Commissioned Articles by Authors/Curators per Author’s expertise on a daily basis

 

Below, see Volume Titles and Cover Pages:

13 LIVE results for Kindle Store: “Aviva Lev-Ari”

 

 

The VOICES of Patients, Hospitals CEOs, Health Care Providers, Caregivers and Families: Personal Experience with Critical Care and Invasive Medical Procedures … E: Patient-Centered Medicine Book 1)

Oct 16, 2017 | Kindle eBook

by Larry H. Bernstein and Aviva Lev-Ari

$0.00

Subscribers read for free.

Read for Free

$49.00$ 49 00 to buyKindle Edition

Get it TODAY, Jan 29

Sold by: Amazon Digital Services LLC

Cancer Therapies: Metabolic, Genomics, Interventional, Immunotherapy and Nanotechnology in Therapy Delivery (Series C Book 2)

May 13, 2017 | Kindle eBook

by Larry H. Bernstein and Demet Sag

$0.00

Subscribers read for free.

Read for Free

$100.00$ 100 00 to buyKindle Edition

Get it TODAY, Jan 29

Sold by: Amazon Digital Services LLC

The Immune System, Stress Signaling, Infectious Diseases and Therapeutic Implications: VOLUME 2: Infectious Diseases and Therapeutics and VOLUME 3: The … (Series D: BioMedicine & Immunology)

Sep 4, 2017 | Kindle eBook

by Larry H. Bernstein and Aviva Lev-Ari

$0.00

Subscribers read for free.

Read for Free

$115.00$ 115 00 to buyKindle Edition

Get it TODAY, Jan 29

Sold by: Amazon Digital Services LLC

Perspectives on Nitric Oxide in Disease Mechanisms (Biomed e-Books Book 1)

Jun 20, 2013 | Kindle eBook

by Margaret Baker PhD and Tilda Barliya PhD

$0.00

Subscribers read for free.

Read for Free

$75.00$ 75 00 to buyKindle Edition

Get it TODAY, Jan 29

5 out of 5 stars 6

Sold by: Amazon Digital Services LLC

Medical Scientific Discoveries for the 21st Century & Interviews with Scientific Leaders (Series E)

Dec 9, 2017 | Kindle eBook

by Larry H. Bernstein and Aviva Lev-Ari

$0.00

Subscribers read for free.

Read for Free

$75.00$ 75 00 to buyKindle Edition

Get it TODAY, Jan 29

Sold by: Amazon Digital Services LLC

Etiologies of Cardiovascular Diseases: Epigenetics, Genetics and Genomics

Nov 28, 2015 | Kindle eBook

by Justin D. Pearlman MD ME PhD MA FACC and Stephen J. Williams PhD

$0.00

Subscribers read for free.

Read for Free

$75.00$ 75 00 to buyKindle Edition

Get it TODAY, Jan 29

Sold by: Amazon Digital Services LLC

Cardiovascular Original Research: Cases in Methodology Design for Content Co-Curation: The Art of Scientific & Medical Curation

Nov 29, 2015 | Kindle eBook

by Larry H. Bernstein MD FCAP and Aviva Lev-Ari PhD RN

$0.00

Subscribers read for free.

Read for Free

$75.00$ 75 00 to buyKindle Edition

Get it TODAY, Jan 29

Sold by: Amazon Digital Services LLC

Medical 3D BioPrinting – The Revolution in Medicine Technologies for Patient-centered Medicine: From R&D in Biologics to New Medical Devices (Series E: Patient-Centered Medicine Book 4)

Dec 30, 2017 | Kindle eBook

by Larry H. Bernstein and Irina Robu

$0.00

Subscribers read for free.

Read for Free

$75.00$ 75 00 to buyKindle Edition

Get it TODAY, Jan 29

Sold by: Amazon Digital Services LLC

Metabolic Genomics & Pharmaceutics (BioMedicine – Metabolomics, Immunology, Infectious Diseases Book 1)

Jul 21, 2015 | Kindle eBook

by Larry H. Bernstein MD FCAP and Prabodah Kandala PhD

$0.00

Subscribers read for free.

Read for Free

$75.00$ 75 00 to buyKindle Edition

Get it TODAY, Jan 29

5 out of 5 stars 1

Sold by: Amazon Digital Services LLC

Cancer Biology and Genomics for Disease Diagnosis (Series C: e-Books on Cancer & Oncology Book 1)

Aug 10, 2015 | Kindle eBook

by Larry H Bernstein MD FCAP and Prabodh Kumar Kandala PhD

$0.00

Subscribers read for free.

Read for Free

$75.00$ 75 00 to buyKindle Edition

Get it TODAY, Jan 29

Sold by: Amazon Digital Services LLC

Genomics Orientations for Personalized Medicine (Frontiers in Genomics Research Book 1)

Nov 22, 2015 | Kindle eBook

by Sudipta Saha PhD and Ritu Saxena PhD

$0.00

Subscribers read for free.

Read for Free

$75.00$ 75 00 to buyKindle Edition

Get it TODAY, Jan 29

Sold by: Amazon Digital Services LLC

Milestones in Physiology: Discoveries in Medicine, Genomics and Therapeutics (Series E: Patient-Centered Medicine Book 3)

Dec 26, 2015 | Kindle eBook

by Larry H. Bernstein MD FACP and Aviva Lev-Ari PhD RN

$0.00

Subscribers read for free.

Read for Free

$75.00$ 75 00 to buyKindle Edition

Get it TODAY, Jan 29

Sold by: Amazon Digital Services LLC

Regenerative and Translational Medicine: The Therapeutic Promise for Cardiovascular Diseases

Dec 26, 2015 | Kindle eBook

by Justin D. Pearlman MD ME PhD MA FACC and Ritu Saxena PhD

$0.00

Subscribers read for free.

Read for Free

$75.00$ 75 00 to buyKindle Edition

Get it TODAY, Jan 29

Sold by: Amazon Digital Services LLC

5        Style Setting: Instruction manuals for Journal, Articles, Books

As BioMed e-Series Editor–in-Chief, Aviva Lev-Ari, PhD, RN was responsible for

·       All the documentation (Instruction manuals) on Style setting, and for

·       Training all team members

·       Journal Articles Format

·       Journal Comment Exchange Format

·       e-Books Production Process:

1.               Volume creation from Journal’s Article Archive,

2.               Format Translation from HTML to .mobi for Kindle devices,

3.               Proof reading process,

4.               Title release,

5.               Book electronic Upload to Amazon.com Cloud.

6.               Connection of all articles and e-Books to Social Media, Ping back generation by mentioning other related articles published in the Journal

 

Lastly, 6, below

6        Annual Workflow Management of Multiple eTOCs – Multi-year Book Publishing Scheduling Plan, 2013 – Present

 

Title Date of Publication Number of Pages
Perspectives on Nitric Oxide in Disease Mechanisms 6/21/2013 895
Cardiovascular Original Research: Cases in Methodology Design for Content Co-Curation 11/30/2015 11039 KB
Etiologies of Cardiovascular Diseases: Epigenetics, Genetics and Genomics 11/29/2015 12333 KB
Regenerative and Translational Medicine: The Therapeutics Promise for Cardiovascular Diseases 12/26/2015 11668 KB
Genomics Orientations for Personalized Medicine 11/23/2015 11724 KB
Cancer Biology & Genomics for Disease Diagnosis 8/11/2015 13744 KB
Cancer Therapies: Metabolic, Genomics, Interventional, Immunotherapy and Nanotechnology in Therapy Delivery 5/18/2017 5408 pages
Metabolic Genomics and Pharmaceutics 7/21/2015 13927 KB
The Immune System, Stress    Signaling, Infectious Diseases and Therapeutic Implications 9/4/2017 3747 pages
The VOICES of Patients, Hospitals CEOs, Health Care Providers, Caregivers and Families: Personal Experience with Critical Care and Invasive Medical Procedures 10/16/2017 826 pages
Medical Scientific Discoveries for the 21st Century & Interviews with Scientific Leaders 12/9/2017 2862 pages
Milestones in Physiology: Discoveries in Medicine, Genomics and Therapeutics 12/27/2015 11125 KB
Medical 3D BioPrinting – The Revolution in Medicine, Technologies for Patient-centered Medicine: From R&D in Biologics to New Medical Devices 12/30/2017 1005 pages
Pharmacological Agents in Treatment of Cardiovascular Disease

 

Work-in-Progress, Expected Publishing date in 2018 ???
Interventional Cardiology and Cardiac Surgery for Disease Diagnosis and Guidance of Treatment Work-in-Progress, Expected Publishing date in 2018

 

???

 

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Novartis’ Kymriah (tisagenlecleucel), FDA approved genetically engineered immune cells, would charge $475,000 per patient, will use Programs that Payers will pay only for Responding Patients

Curator: Aviva Lev-Ari, PhD, RN

 

UPDATED on 9/1/2017:

This Pioneering $475,000 Cancer Drug Comes With A Money-Back Guarantee

Novartis defends the eye-popping price of its pioneering gene therapy with arguments about its $1 billion expenditure—and novel “value-based” pricing.

https://www.fastcompany.com/40461214/how-novartis-is-defending-the-record-475000-price-of-its-pioneering-gene-therapy-cancer-drug-car-t-kymriah

 

On 8/30/2017 we wrote:

FDA has approved the world’s first CAR-T therapy, Novartis for Kymriah (tisagenlecleucel) and Gilead’s $12 billion buy of KitePharma, no approved drug and Canakinumab for Lung Cancer (may be?)

Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2017/08/30/fda-has-approved-the-worlds-first-car-t-therapy-novartis-for-kymriah-tisagenlecleucel-and-gileads-12-billion-buy-of-kite-pharma-no-approved-drug-and-canakinumab-for-lung-cancer-may-be/

 

The Price for the Treatment was published on 8/31/2017, a Value-based Pricing Payment Model of a $475,000 per patient charge for the responding patients after ONE month of treatment. Novartis says it takes an average of 22 days to create the therapy, from the time a patient’s cells are removed to when they are infused back into the patient. Kymriah will initially be available at 20 U.S. hospitals within a month, Novartis says. Eventually, 32 total sites will offer the therapy. 

CAR-T gained national attention three years ago when Carl June, a researcher at the University of Pennsylvania, used to put a young girl’s acute lymphoblastic leukemia. Genetically altering the girl’s immune cells had made her deathly ill, but June had used a Roche drug, Actemra, to treat the side effects. She lived, and the results were published in The New England Journal of Medicine. Novartis bought the rights to the Penn treatment for just $20 million up front.

Pharma Buying the right to use from an Academic Institution is a known route to leap frog the R&D lengthy process of Drug discovery.

“I’ve told the team that resources are not an issue. Speed is the issue,” says Novartis’ Chief Executive Joseph Jimenez, told Forbes in a cover story about the work then.

The FDA calls this CAR-T therapy treatment, made by Novartis, the “first gene therapy” in the U.S. The therapy is designed to treat an often-lethal type of blood and bone marrow cancer that affects children and young adults. The FDA defines gene therapy as a medicine that “introduces genetic material into a person’s DNA to replace faulty or missing genetic material” to treat a disease or medical condition. This is the first such therapy to be available in the U.S., according to the FDA.

Two gene therapies for rare, inherited diseases have already been approved in Europe.

To further evaluate the long-term safety, Novartis is also required to conduct a post-marketing observational study involving patients treated with Kymriah.

The FDA granted Kymriah Priority Review and Breakthrough Therapy designations. The Kymriah application was reviewed using a coordinated, cross-agency approach. The clinical review was coordinated by the FDA’s Oncology Center of Excellence, while CBER conducted all other aspects of review and made the final product approval determination.

The FDA granted approval of Kymriah to Novartis Pharmaceuticals Corp. The FDA granted the expanded approval of Actemra to Genentech Inc.

FDA commissioner Scott Gottlieb in a statement.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” 

“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”

https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm

The Protocol

A patient’s T cells are extracted and cryogenically frozen so that they can be transported to Novartis’s manufacturing center in New Jersey. There, the cells are genetically altered to have a new gene that codes for a protein—called a chimeric antigen receptor, or CAR. This protein directs the T cells to target and kill leukemia cells with a specific antigen on their surface. The genetically modified cells are then infused back into the patient.

In a clinical trial of 63 children and young adults with a type of acute lymphoblastic leukemia, 83 percent of patients that received the CAR-T therapy had their cancers go into remission within three months. At six months, 89 percent of patients who received the therapy were still living, and at 12 months, 79 percent had survived.

https://www.technologyreview.com/s/608771/the-fda-has-approved-the-first-gene-therapy-for-cancer/?utm_campaign=add_this&utm_source=email&utm_medium=post

CAR-T Therapies: Product/Molecules/MOA under Development:

  • Similar CAR-T treatments were being developed at other institutions including
  • Memorial Sloan-Kettering Cancer Center,
  • Seattle Children’s Hospital, and
  • The National Cancer Institute.
  • The Memorial and Seattle work was spun off into a startup called Juno Therapeutics, which has fallen behind. Juno Therapeutics ended a CAR-T study earlier this year after patients died from cerebral edema, or swelling in the brain.
  • The NCI work became the basis for the product being developed by Kite Pharma. Kite Pharma, which is awaiting FDA approval for its CAR-T therapy to treat a form of blood cancer in adults, was this week bought out by Gilead in a deal worth $11.9 billion.

On Cambridge Healthtech Institute’s 4th Annual Adoptive T Cell Therapy, Delivering CAR, TCR, and TIL from Research to Reality, August 29 – 30, 2017 | Sheraton Boston | Boston, MA

TUESDAY, AUGUST 29 – I covered in Real Time the talk on Juno Therapeutics: Building Better T Cell Therapies: The Power of Molecular Profiling by Mark Bonyhadi, Ph.D., Head, Research and Academic Affairs, Juno Therapeutics

https://pharmaceuticalintelligence.com/2017/08/29/live-829-chis-oncolytic-virus-immunotherapy-and-adoptive-cell-therapy-august-28-29-2017-sheraton-boston-hotel-boston-ma/

 

Precision Medicine is Costly and not a Rapid manufacturing process

All of the CAR-T products are expensive to make, and must be manufactured on an individual basis for each new patient from the patient’s own T-cells, a type of white blood cells, a process that takes weeks.

  • How quickly companies can speed up manufacturing.
  • Kymriah will be manufactured at a facility in Morris Plains, N.J.
  • CAR-T technology, which has so far been used only in patients with blood cancers that have not been cured by other treatments, can be used earlier in the disease or for solid tumors: Breast, Prostate, Melanomas.

https://www.forbes.com/sites/matthewherper/2017/08/30/fda-approves-novartis-treatment-that-alters-patients-cells-to-fight-cancer/#2aecb25b4400

Prediction How Patients will Far Well – Researchers use a big-data approach to find links between different genes and patient survival.

https://www.technologyreview.com/s/608666/a-cancer-atlas-to-predict-how-patients-will-fare/?set=

A pathology atlas of the human cancer transcriptome

+ See all authors and affiliations

Science  18 Aug 2017:
Vol. 357, Issue 6352, eaan2507
DOI: 10.1126/science.aan2507

Modeling the cancer transcriptome

Recent initiatives such as The Cancer Genome Atlas have mapped the genome-wide effect of individual genes on tumor growth. By unraveling genomic alterations in tumors, molecular subtypes of cancers have been identified, which is improving patient diagnostics and treatment. Uhlen et al. developed a computer-based modeling approach to examine different cancer types in nearly 8000 patients. They provide an open-access resource for exploring how the expression of specific genes influences patient survival in 17 different types of cancer. More than 900,000 patient survival profiles are available, including for tumors of colon, prostate, lung, and breast origin. This interactive data set can also be used to generate personalized patient models to predict how metabolic changes can influence tumor growth.

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FDA has approved the world’s first CAR-T therapy, Novartis for Kymriah (tisagenlecleucel) and Gilead’s $12 billion buy of Kite Pharma, no approved drug and Canakinumab for Lung Cancer (may be?)

Curator: Aviva Lev-Ari, PhD, RN

UPDATED on 9/7/2017

Here’s the inside account of Gilead’s 11-week sprint to its $12B Kite buyout – ENDPOINTS NEWS

UPDATED on 8/31/2017

Gilead-Kite: A New Transformative Deal For Biotech, AUG 30, 2017

Gilead has made a big bet on new technology in Kite’s immunotherapy platforms and has reduced the number of credible large players in the space.

With a reputation for intense diligence and dynamism in its business development efforts, Gilead’s management team will only bolster the immunotherapy field as it prepares to face off with Novartis, its immediate competitor, and enters squarely in the province of Merck and Bristol Myers Squibb, two of the leaders in immuno-oncology.

Gilead has reinvented the transformative transaction for the sector.

https://www.forbes.com/sites/stephenbrozak/2017/08/30/gilead-kite-a-new-transformative-deal-and-maybe-the-new-future-of-healthcare-deals/#fc64fca65d49

 

I attended this week the Cambridge Healthtech Institute’s 4th Annual

Adoptive T Cell Therapy

Delivering CAR, TCR, and TIL from Research to Reality
August 29 – 30, 2017 | Sheraton Boston | Boston, MA

 

The following talks on 8/29/2017 presented the frontier of CAR-T Therapies and Technologies from lab to bed side:

  • Building Better T Cell Therapies: The Power of Molecular Profiling

Mark Bonyhadi, Ph.D., Head, Research and Academic Affairs, Juno Therapeutics

  • Tricked-Out Cars, the Next Generation of CAR T Cells

Richard Morgan, Ph.D., Vice President, Immunotherapy, Bluebird Bio

  • The Generation of Lentiviral Vector-Modified CAR-T Cells Using an Automated Process

Boro Dropulic, Ph.D., General Manager and CSO, Lentigen Technology, Inc.

I covered this event in Real Time for the Press

LIVE – 8/29 – CHI’s Oncolytic Virus Immunotherapy and ADOPTIVE CELL THERAPY, August 28-29, 2017 Sheraton Boston Hotel | Boston, MA

https://pharmaceuticalintelligence.com/2017/08/29/live-829-chis-oncolytic-virus-immunotherapy-and-adoptive-cell-therapy-august-28-29-2017-sheraton-boston-hotel-boston-ma/

 

One year ago we published the following:

What does this mean for Immunotherapy? FDA put a temporary hold on Juno’s JCAR015, Three Death of Celebral Edema in CAR-T Clinical Trial and Kite Pharma announced Phase II portion of its CAR-T ZUMA-1 trial

https://pharmaceuticalintelligence.com/2016/07/09/what-does-this-mean-for-immunotherapy-fda-put-a-temporary-hold-on-jcar015-three-death-of-celebral-edema-in-car-t-clinical-trial-and-kite-pharma-announced-phase-ii-portion-of-its-car-t-zuma-1-trial/

 

SOURCE

Is Canakinumab the Next Viagra?

In this Revolution and Revelation, Milton Packer explains how safety data can sometimes trump a primary endpoint

by Milton PackerAugust 30, 2017

https://www.medpagetoday.com/Blogs/RevolutionandRevelation/67605

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Genomic Diagnostics: Three Techniques to Perform Single Cell Gene Expression and Genome Sequencing Single Molecule DNA Sequencing

Curator: Aviva Lev-Ari, PhD, RN

 

This article presents Three Techniques to Perform Single Cell Gene Expression and Genome Sequencing Single molecule DNA sequencing

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Ido Sagi – PhD Student @HUJI, 2017 Kaye Innovation Award winner for leading research that yielded the first successful isolation and maintenance of haploid embryonic stem cells in humans.

Reporter: Aviva Lev-Ari, PhD, RN

 

Ido Sagi – PhD Student, Silberman Institute of Life Sciences, HUJI, Israel

  • Ido Sagi’s research focuses on studying genetic and epigenetic phenomena in human pluripotent stem cells, and his work has been published in leading scientific journals, including NatureNature Genetics and Cell Stem Cell.
  • Ido Sagi received BSc summa cum laude in Life Sciences from the Hebrew University, and currently pursues a PhD at the laboratory of Prof. Nissim Benvenisty at the university’s Department of Genetics in the Alexander Silberman Institute of Life Sciences.

The Kaye Innovation Awards at the Hebrew University of Jerusalem have been awarded annually since 1994. Isaac Kaye of England, a prominent industrialist in the pharmaceutical industry, established the awards to encourage faculty, staff and students of the Hebrew University to develop innovative methods and inventions with good commercial potential, which will benefit the university and society.

Publications – Ido Sagi

Comparable frequencies of coding mutations and loss of imprinting in human pluripotent cells derived by nuclear transfer and defined factors.
Cell Stem Cell 2014 Nov 6;15(5):634-42. Epub 2014 Nov 6.
The New York Stem Cell Foundation Research Institute, New York, NY 10032, USA; Naomi Berrie Diabetes Center & Department of Pediatrics, College of Physicians and Surgeons, Columbia University, New York, NY 10032, USA. Electronic address:

November 2014

 



Stem cells: Aspiring to naivety.
Nature 2016 12 30;540(7632):211-212. Epub 2016 Nov 30.
The Azrieli Center for Stem Cells and Genetic Research, Department of Genetics, Silberman Institute of Life Sciences, The Hebrew University of Jerusalem, Jerusalem 91904, Israel.
November 2016

Download Full Paper

SOURCE

Other related articles on Genetic and Epigenetic phenomena in human pluripotent stem cells published by LPBI Group can be found in the following e-Books on Amazon.com

e-Books in Medicine

https://www.amazon.com/s/ref=dp_byline_sr_ebooks_9?ie=UTF8&text=Aviva+Lev-Ari&search-alias=digital-text&field-author=Aviva+Lev-Ari&sort=relevancerank

9 results for Kindle Store : “Aviva Lev-Ari”

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    Etiologies of Cardiovascular Diseases: Epigenetics, Genetics and Genomics

    Nov 28, 2015 | Kindle eBook

    by Justin D. Pearlman MD ME PhD MA FACC and Stephen J. Williams PhD
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    Cancer Therapies: Metabolic, Genomics, Interventional, Immunotherapy and Nanotechnology in Therapy Delivery (Series C Book 2)

    May 13, 2017 | Kindle eBook

    by Larry H. Bernstein and Demet Sag
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    Perspectives on Nitric Oxide in Disease Mechanisms (Biomed e-Books Book 1)

    Jun 20, 2013 | Kindle eBook

    by Margaret Baker PhD and Aviva Lev-Ari PhD RN
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    Cancer Biology and Genomics for Disease Diagnosis (Series C: e-Books on Cancer & Oncology Book 1)

    Aug 10, 2015 | Kindle eBook

    by Larry H Bernstein MD FCAP and Prabodh Kumar Kandala PhD
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    Genomics Orientations for Personalized Medicine (Frontiers in Genomics Research Book 1)

    Nov 22, 2015 | Kindle eBook

    by Sudipta Saha PhD and Ritu Saxena PhD
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    Metabolic Genomics & Pharmaceutics (BioMedicine – Metabolomics, Immunology, Infectious Diseases Book 1)

    Jul 21, 2015 | Kindle eBook

    by Larry H. Bernstein MD FCAP and Prabodah Kandala PhD
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    Milestones in Physiology: Discoveries in Medicine, Genomics and Therapeutics (Series E: Patient-Centered Medicine Book 3)

    Dec 26, 2015 | Kindle eBook

    by Larry H. Bernstein MD FACP and Aviva Lev-Ari PhD RN
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    Regenerative and Translational Medicine: The Therapeutic Promise for Cardiovascular Diseases

    Dec 26, 2015 | Kindle eBook

    by Justin D. Pearlman MD ME PhD MA FACC and Ritu Saxena PhD
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    Cardiovascular Original Research: Cases in Methodology Design for Content Co-Curation: The Art of Scientific & Medical Curation

    Nov 29, 2015 | Kindle eBook

    by Larry H. Bernstein MD FCAP and Aviva Lev-Ari PhD RN
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