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Posts Tagged ‘Weill Cornell Medical College of Cornell University’


Precision Medicine: The Future of Medicine?

Reporter: Aviva Lev-Ari, PhD, RN

Dr. Laurie Glimcher, dean of Weill Cornell Medical College, and Dr. Robert Langer, the Koch Institute Professor at MIT, talk to the “CBS This Morning” co-hosts about what’s next in the fight against diseases like Alzheimer’s, cancer, and diabetes.

VIEW VIDEO

http://www.cbsnews.com/video/watch/?id=50149783n

Free Webinar:

The Economics of Precision Medicine: 

How Personalizing Treatment can Bend the Cost Curve by 

Improving the Value Delivered by Healthcare Innovations

In a world where it is clear that healthcare costs must be contained, how can we afford to pay for innovation? This webinar will explore how personalizing treatment can offer an escape from the innovation-cost conundrum. By simultaneously increasing clinical development efficiency and the treatment effectiveness, targeting clinical innovations to the patients most likely to benefit can improve healthcare value per dollar spent while maintaining the ROI levels needed to support investment in innovation. We believe precision medicine should play a more prominent role in the cost containment discussion of healthcare reform.

By attending this Webinar, you will learn how to:

Help clients develop product development and commercialization strategies that get leverage from the benefits of precision medicine 

Support positioning of innovations as part of the healthcare solution, not the problem 

Understand and communicate the value proposition of precision medicine for payers, government decision makers, and legislators

The Economics of Precision Medicine: How Personalizing Treatment can Bend the Cost Curve by Improving the Value Delivered by Healthcare Innovations

Thursday, July 25, 2013

11:30 am PDT / 2:30 pm EDT

1 hour

Who should attend:

Franchise and Marketing Leaders

Therapeutic Area Leads

Medical Affairs

Government Affairs/Public Policy

Health Economics and Market Access

Webinar agenda:

Is the high cost of healthcare innovation incompatible with control of healthcare costs?

Cost-effectiveness criteria and how they can be met

Taking cost out of clinical development

Case Example: How everyone can win

Practical impact on development and commercialization strategies

Q&A

Speaker information: 

David Parker, Ph.D., Vice President, Market Access Strategy, Precision for Medicine

Vicki L. Seyfert-Margolis, Chief Scientific and Strategy Officer, Precision for Medicine

Harry Glorikian, Managing Director, Strategy, Precision for Medicine

Cambridge Healthtech Institute, 250 First Avenue, Suite 300, Needham, MA 02494

Tel: 781-972-5400 | Fax: 781-972-5425

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Reporter: Aviva Lev-Ari, PhD, RN

Scar Tissue In Damaged Hearts Reprogrammed By Gene Therapy Into Healthy Heart Muscle

Article Date: 08 Jan 2013 – 0:00 PST

A cocktail of three specific genes can reprogram cells in the scars caused by heart attacks into functioning muscle cells, and the addition of a gene that stimulates the growth of blood vessels enhances that effect, said researchers from Weill Cornell Medical College, Baylor College of Medicine and Stony Brook University Medical Center in a report that appears online in the Journal of the American Heart Association. 

“The idea of reprogramming scar tissue in the heart into functioning heart muscle was exciting,” said Dr. Todd K. Rosengart, chair of the Michael E. DeBakey Department of Surgery at BCM and the report’s corresponding author. “The theory is that if you have a big heart attack, your doctor can just inject these three genes into the scar tissue during surgery and change it back into heart muscle. However, in these animal studies, we found that even the effect is enhanced when combined with the VEGF gene.” 

“This experiment is a proof of principle,” said Dr. Ronald G. Crystal, chairman and professor of genetic medicine at Weill Cornell Medical College and a pioneer in gene therapy, who played an important role in the research. “Now we need to go further to understand the activity of these genes and determine if they are effective in even larger hearts.” 

During a heart attack, blood supply is cut off to the heart, resulting in the death of heart muscle. The damage leaves behind a scar and a much weakened heart. Eventually, most people who have had serious heart attacks will develop heart failure

Changing the scar into heart muscle would strengthen the heart. To accomplish this, during surgery, Rosengart and his colleagues transferred three forms of the vascular endothelial growth factor (VEGF) gene that enhances blood vessel growth or an inactive material (both attached to a gene vector) into the hearts of rats. Three weeks later, the rats received either Gata4, Mef 2c and Tbx5 (the cocktail of transcription factor genes called GMT) or an inactive material. (A transcription factor binds to specific DNA sequences and starts the process that translates the genetic information into a protein.) 

The GMT genes alone reduced the amount of scar tissue by half compared to animals that did not receive the genes, and there were more heart muscle cells in the animals that were treated with GMT. The hearts of animals that received GMT alone also worked better as defined by ejection fraction than those who had not received genes. (Ejection fraction refers to the percentage of blood that is pumped out of a filled ventricle or pumping chamber of the heart.) 

The hearts of the animals that had received both the GMT and the VEGF gene transfers had an ejection fraction four times greater than that of the animals that had received only the GMT transfer. 

Rosengart emphasizes that more work needs to be completed to show that the effect of the VEGF is real, but it has real promise as part of a new treatment for heart attack that would minimize heart damage. 

“We have shown both that GMT can effect change that enhances the activity of the heart and that the VEGF gene is effective in improving heart function even more,” said Dr. Crystal. 

The idea started with the notion of induced pluripotent stem cells – reprograming mature specialized cells into stem cells that are immature and can differentiate into different specific cells needed in the body. Dr. Shinya Yamanaka and Sir John B. Gurdon received the Nobel Prize in Medicine and Physiology for their work toward this goal this year. 

However, use of induced pluripotent stem cells has the potential to cause tumors. To get around that, researchers in Dallas and San Francisco used the GMT cocktail to reprogram the scar cells into cardiomyocytes (cells that become heart muscle) in the living animals. 

Now Rosengart and his colleagues have gone a step farther – encouraging the production of new blood vessels to provide circulation to the new cells.

REFERENCES:

Others who took part in this work include Megumi Mathison, Ronald Gersch, Ahmed Nasser, Sarit Lilo, Mallory Korman, Mitchell Fourman, Kenneth Shroyer, Jianchang Yang, Yupo Ma, all of Stony Brook University Medical Center and Neil Hackett of Weill Cornell Medical College.
Funding for this work came from the generosity of James and Lisa Cohen.
Weill Cornell Medical College

CITATIONS:

MLA

n.p. “Scar Tissue In Damaged Hearts Reprogrammed By Gene Therapy Into Healthy Heart Muscle.” Medical News Today. MediLexicon, Intl., 8 Jan. 2013. Web.
9 Jan. 2013. <http://www.medicalnewstoday.com/releases/254618.php>

APA

n.p. (2013, January 8). “Scar Tissue In Damaged Hearts Reprogrammed By Gene Therapy Into Healthy Heart Muscle.” Medical News Today. Retrieved from
http://www.medicalnewstoday.com/releases/254618.php.

SOURCE:

http://www.medicalnewstoday.com/releases/254618.php 

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Reporter: Aviva Lev-Ari, PhD, RN

 

Gates Foundation funds research to improve health in developing countries
Lauren Braun as a volunteer in Peru

Division of Nutritional Sciences
As a volunteer in the summer of 2008, Lauren Braun ’11 fills a prescription in a makeshift rural pharmacy in Peru.
Alma Sana bracelets

Provided/Alma Sana
Alma Sana bracelets use symbols to avoid language barriers.

A Cornell plant virologist, an alumna and three Weill Cornell Medical College researchers have each received grants from the Bill & Melinda Gates Foundation‘s Grand Challenges in Global Health initiative.

One grant awarded to Jeremy Thompson in the Department of Plant Pathology and Plant-Microbe Biology will fund a project that takes advantage of new technology to rapidly determine the structure of RNA in viruses, which may lead to a new method for developing virus-resistant plants. Thompson, a research associate in the lab of Keith Perry, associate professor of plant pathology, will work with Perry to uncover new targets for plant virus resistance and with Julius Lucks, assistant professor of chemical and biomolecular engineering, who has developed new RNA structure mapping technology.

Viruses are known to use their RNA to hijack the replication machinery in host cells to make more copies of the virus. The researchers hope that determining the RNA structure will reveal plant proteins that are involved in viral replication.

“We want to try and map the structure of viral RNA, map the way it folds, and then we can potentially identify host proteins that are involved in virus replication and function,” said Thompson.

Once these plant proteins are identified, the researchers will look for genes that code for those proteins and try to alter their expression within the plant. “If we can affect the amount of protein involved, we can potentially hinder virus replication,” Thompson added. Using refined engineering methods to knock out or silence such protein-coding genes, the researchers may then create lines of virus-resistant plants.

The researchers will begin by examining viruses and host proteins in bean, tobacco and arabidopsis; bean, because of its importance as a staple in developing countries and the latter two because their genomes have been fully sequenced.

The one-and-a-half year, $100,000 grant represents a first phase that, if successful, allows the team to become eligible for phase two and an additional $1 million.

Lauren Braun

Braun

As the main objective of the Gates Foundation Grand Challenges in Global Health initiative is to improve the quality of life in developing countries, this project aims to “improve resistance against particular diseases for small-holder farmers, with all intellectual property being open to developing countries,” Thompson said. Plant viruses lead to billions of dollars in agricultural production losses each year.

Lauren Braun ’11 received a $100,000 grant to field-test in Peru a simple, inexpensive immunization-tracking bracelet for babies. Braun conceived the idea after spending the summer of 2008 as a volunteer at two rural health clinics in Peru, and she presented it on campus in the Entrepreneurship@Cornell’s 2011 Big Idea Competition.

The World Health Organization estimates that globally 1.5 million children die of vaccine-preventable diseases each year, and one in five children will die from such a disease before age 5.

Braun formed the nonprofit Alma Sana Inc. (Spanish for healthy soul) to manufacture and distribute the bracelets, which bypass language barriers and illiteracy by using symbols to show mothers the vaccinations their children need and numbers to show when they are due. The bracelet is to be worn by a child from birth to age 4, with the goal that more children will live to age 5.

A paper reminder system failed, Braun reports, because children are not brought in for their vaccinations and stored vaccine spoils and must be discarded, increasing costs. The bracelet also tells public health workers which vaccination each child needs.

The Gates Foundation initiative seeks new approaches to optimize immunization systems. In 2010, they said, a quarter of a million doses of pentavalent vaccine, costing nearly $1 million, expired in one country’s central store because the system charged with delivering them was not ready to manage it.

Three researchers at Weill Cornell Medical College have received Gates Foundation grants totaling $1.5 million from the Grand Challenges initiative for innovative research aimed at fighting HIV and tuberculosis.

SOURCE:

 

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Reporter: Aviva Lev-Ari, PhD, RN

Public release date: 18-Oct-2012
Contact: Lauren Woods
law2014@med.cornell.edu
212-821-0560
New York- Presbyterian Hospital/Weill Cornell Medical Center/Weill Cornell Medical College

 

New study shows reprogrammed amniotic fluid cells could treat vascular diseases

Weill Cornell Researchers discover a new effective approach for converting amniotic fluid-derived cells into endothelial cells to repair damaged blood vessels in heart disease, stroke, diabetes and trauma

NEW YORK (Oct. 18, 2012) — A research team at Weill Cornell Medical College has discovered a way to utilize diagnostic prenatal amniocentesis cells, reprogramming them into abundant and stable endothelial cells capable of regenerating damaged blood vessels and repairing injured organs.

Their study, published online today in Cell, paints a picture of a future therapy where amniotic fluid collected from thousands of amniocentesis procedures yearly, during mid-pregnancy to examine fetal chromosomes, would be collected with the permission of women undergoing the test. These cells, which are not embryonic, would then be treated with a trio of genes that reprogram them quickly into billions of endothelial cells — the cells that line the entire circulatory system. The new endothelial cells could be frozen and banked the same way blood is, and patients in need of blood vessel repair would be able to receive the cells through a simple injection.

If proven in future studies, this novel therapy could dramatically improve treatment for disorders linked to a damaged vascular system, including heart disease, stroke, lung diseases such as emphysema, diabetes, and trauma, says the study’s senior investigator, Dr. Shahin Rafii, the Arthur B. Belfer Professor in Genetic Medicine at Weill Cornell Medical College and co-director of its Ansary Stem Cell Institute.

“Currently, there is no curative treatment available for patients with vascular diseases, and the common denominator to all these disorders is dysfunction of blood vessels, specifically endothelial cells that are the building blocks of the vessels,” says Dr. Rafii, who is also a Howard Hughes Medical Institute investigator.

But these cells do much more than just provide the plumbing to move blood. Dr. Rafii has recently led a series of transformative studies that show endothelial cells in blood vessels produce growth factors that actively participate in organ maintenance, repair and regeneration. So while damaged vessels cannot repair the organs they nurture with blood, he says an infusion of new endothelial cells could.

“Replacement of the dysfunctional endothelial cells with transplantation of normal, properly engineered cultured endothelial cells could potentially provide for a novel therapy for many patients,” says study co-author Dr. Sina Rabbany, adjunct associate professor of bioengineering in genetic medicine at Weill Cornell. “In order to engineer tissues with clinically relevant dimensions, endothelial cells can be assembled into porous three-dimensional scaffolds that, once introduced into a patient’s injured organ, could form true blood vessels.”

Dr. Rafii says that this study will potentially create a new field of translational vascular medicine. He estimates that as few as four years are needed for the preclinical work to seek FDA approval to start human clinical trials to advance the potential of reprogrammed endothelial cells for treatment of vascular disorders.

As part of their study, the research team proved, in mice, that endothelial cells reprogrammed from human amniotic cells could engraft into an injured liver to form stable, normal and functional blood vessels. “We have shown that these engrafted endothelial cells have the capacity to produce unique growth factors to promote regeneration of the liver cells,” says the study’s lead investigator, Dr. Michael Ginsberg, a senior postdoctoral associate in Dr. Rafii’s laboratory.

“The novelty of this technique is that, from 100,000 amniotic cells — a small amount — we grew more than six billion new authentic endothelial cells within a matter of weeks,” Dr. Ginsberg says. “And when we injected these cells into mice, a substantial amount of them engrafted into regenerating vessels. It was remarkable to see that these cells went right to work building new blood vessels in the liver as well as producing the right growth factors that could potentially regenerate and repair injured organs.”

The Goldilocks of Cellular Reprogramming

To date, there have been many failed attempts to clinically produce endothelial cells that can be used to treat patients. Isolation of endothelial cells from adult organs so they can be grown in the laboratory is not efficient, according to Dr. Daylon James, study co-author and an assistant professor of stem cell biology in reproductive medicine at Weill Cornell Medical College. Attempts to produce the cells from the body’s master pluripotent stem cells have also not worked out. Experiments have shown that prototypical pluripotent stem cells, such as embryonic stem cells, which have the potential to become any cell in the body, produce endothelial cells but often grow poorly, and if not fully differentiated could potentially cause cancer. “Coaxing adult cells to revert to a stem-like state so they can then be pushed to form endothelial cells is, at this point, not clinically feasible, and ongoing studies in my lab are focused on achieving this goal,” says Dr.

James, who is also assistant professor of stem cell biology in obstetrics and gynecology and genetic medicine at Weill Cornell. Therefore, Dr. Rafii’s team searched for a new source of cells that they could turn into a vast supply of stable endothelial cells. They probed human amniotic fluid-derived cells, which some studies had suggested have the potential to become differentiated cell types, if stimulated in the right way — which no one had yet identified.

In their first experiments with these cells three years ago, Dr. Ginsberg used cells taken from an amniocentesis given at 16 weeks of gestation. Researchers found that amniotic cells are the “Goldilocks” of cellular programming. “They are not as plastic and unstable as endothelial cells derived from embryonic cells or as stubborn as those produced from reprogramming differentiated adult cells,” Dr. Ginsberg says. Instead, he says amniotic cells provide conditions that are just right — the so-called “Goldilocks Principle” — for producing endothelial cells.

But in order to make that discovery, the researchers had to know how to reprogram the amniotic cells. To this end, they looked for the genes that embryonic stem cells use to differentiate into endothelial cells. Dr. Rafii’s group identified three genes that are expressed during vascular development, all of which are members of the E-twenty six (ETS) family of transcription factors known to regulate cellular differentiation, especially blood vessel formation.

Next, they used gene transfer technology to insert the three genes into mature amniotic cells, and then shut one of them off after a brief and critical period of activity by using a special molecular inhibitor. Remarkably, 20 percent of the amniotic cells could efficiently be reprogrammed into endothelial cells. “This is quite an achievement since current strategies to reprogram adult cells result less than one percent of the time in successful reprogramming into endothelial cells,” says Dr. Rafii.

“These transcription factors do not cause cancer, and the endothelial cells reprogrammed from human amniotic cells are not tumorigenic and could in the future be infused into patients with a large margin of safety,” Dr. Ginsberg says.

The findings suggest that other transcription factors could be used to reprogram the amniotic cells into many other tissue-specific cells, such as those that make up muscles, the brain, pancreatic islet cells and other parts of the body.

“While our work focused primarily on the reprogramming of amniotic cells into endothelial cells, we surmise that through the use of other transcription factors and growth conditions, our group and others will be able to reprogram mouse and human amniotic cells virtually into every organ cell type, such as hepatocytes in the liver, cardiomyocytes in heart muscle, neurons in the brain and even chondrocytes in cartilage, just to name a few,” Dr. Ginsberg says.

“Obviously, the implications of these findings would be enormous in the field of translational regenerative medicine,” emphasizes study co-author Dr. Zev Rosenwaks, the Revlon Distinguished Professor of Reproductive Medicine in Obstetrics and Gynecology at Weill Cornell Medical College and director and physician-in-chief of the Ronald O. Perelman and Claudia Cohen Center for Reproductive Medicine at NewYork-Presbyterian Hospital/Weill Cornell Medical Center. “The greatest obstacle to overcome in the pursuit to regenerate specific tissues and organs is the requirement for substantial levels of cells — in the billions — that are stable, safe and durable. Our approach will bring us closer to this milestone.”

“Most importantly, these endothelial cells could be reprogrammed from amniotic cells from genetically diverse individuals,” says co-author Dr. Venkat R. Pulijaal, director of the Cytogenetic Laboratory, associate professor of clinical pathology and laboratory medicine at Weill Cornell. What endothelial cells a patient receives would depend on their human leukocyte antigen (HLA) type, which is a set of self-recognition molecules that enable doctors to match a patient with potential donors of blood or tissue.

“Selecting the proper immunologically matched endothelial cells for each patient would be akin to blood typing. There are only so many varieties, which are well represented across the amniotic fluid cells that could be obtained, frozen and banked from wide variety of ethnic groups around the world,” Dr. Rafii says.

A patent has been filed on the discovery.

 

Other study co-authors from Weill Cornell Medical College include: Dr. Bi-Sen Ding, Dr. Daniel Nolan, Dr. Fuqiang Geng, Dr. Jason M. Butler, Dr. William Schachterle, Dr. Susan Mathew, Dr. Stephen T. Chasen, Dr. Jenny Xiang, Dr. Koji Shido and Dr. Olivier Elemento.

Dr. Rafii’s research is funded by the Howard Hughes Medical Institute, the National Heart, Lung, and Blood Institute, the Ansary Stem Cell Institute at Weill Cornell Medical College, the Empire State Stem Cell Board and New York State Department of Health grants, and the Qatar National Priorities Research Foundation.

Weill Cornell Medical College

Weill Cornell Medical College, Cornell University’s medical school located in New York City, is committed to excellence in research, teaching, patient care and the advancement of the art and science of medicine, locally, nationally and globally. Physicians and scientists of Weill Cornell Medical College are engaged in cutting-edge research from bench to bedside, aimed at unlocking mysteries of the human body in health and sickness and toward developing new treatments and prevention strategies. In its commitment to global health and education, Weill Cornell has a strong presence in places such as Qatar, Tanzania, Haiti, Brazil, Austria and Turkey. Through the historic Weill Cornell Medical College in Qatar, the Medical College is the first in the U.S. to offer its M.D. degree overseas. Weill Cornell is the birthplace of many medical advances — including the development of the Pap test for cervical cancer, the synthesis of penicillin, the first successful embryo-biopsy pregnancy and birth in the U.S., the first clinical trial of gene therapy for Parkinson’s disease, and most recently, the world’s first successful use of deep brain stimulation to treat a minimally conscious brain-injured patient. Weill Cornell Medical College is affiliated with NewYork-Presbyterian Hospital, where its faculty provides comprehensive patient care at NewYork-Presbyterian Hospital/Weill Cornell Medical Center. The Medical College is also affiliated with the Methodist Hospital in Houston. For more information, visit weill.cornell.edu.

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Reporter: Aviva Lev-Ari, PhD, RN

A pivotal study of a third drug will end later this year, and results from a small, early test of it will be reported next week at an Alzheimer’s conference in Vancouver, British Columbia.

These three treatments are practically the “last men standing” in late-stage trials, after more than a decade of failed efforts to develop a drug to halt the mind-robbing disease. Current medicines such as Aricept and Namenda just temporarily ease symptoms. There is no known cure.

Experts say that if these fail, drug companies may pull out of the field in frustration, leaving little hope for the millions of people with the disease. An estimated 35 million people worldwide have dementia, which includes Alzheimer’s. In the U.S., experts say about 5 million have Alzheimer’s.

http://www.timesleader.com/stories/Last-drugs-standing,176933#ixzz20uq13yCg

http://www.timesleader.com/stories/Last-drugs-standing,176933

The three drugs and their developers are:

• Bapineuzumab (bap-ih-NOOZ-uh-mab), by Pfizer Inc. and Johnson & Johnson’s Janssen Alzheimer Immunotherapy unit.

Solanezumab (sol-ah-NAYZ-uh-mab), by Eli Lilly & Co.- Antibody

• Gammagard, by Baxter International Inc. – IV Immune Globulin

http://www.timesleader.com/stories/Last-drugs-standing,176933#ixzz20ulwcTEP

All are given as periodic intravenous infusions; some companies are trying to reformulate them so they could be given as shots. If a major study shows that one of the drugs works, there will be a huge effort to make it more convenient and practical, Thies predicted.

Still, it would probably be very expensive.

The first two on the list are lab-made, single antibodies against amyloid. Gammagard is intravenous immune globulin, or IVIG — multiple, natural antibodies culled from blood. Half a dozen companies already sell IVIG to treat immune system and blood disorders. It takes 130 plasma donations to make enough to treat one patient for a year.

Treating Alzheimer’s with IVIG would cost $2,000 to $5,000 every two weeks, depending on the patient’s weight, said Dr. Norman Relkin, head of a memory disorders program at New York-Presbyterian Hospital/Weill Cornell Medical Center. He consults for some drugmakers and has patents for tests that measure amyloid.

http://www.timesleader.com/stories/Last-drugs-standing,176933#ixzz20uoQU79G

Concern arose when an earlier study found possible bleeding or brain abnormalities in up to 10 percent of patients on the drug. However, most had no symptoms and were able to resume treatment after a brief break, Yuen said. In fact, some researchers think these changes might be a sign the drug is working to clear the amyloid plaque.

The fact that independent monitors have not stopped the new studies has made Dr. Reisa Sperling optimistic the drug will prove to be safe. Director of the Alzheimer’s center at Brigham and Women’s Hospital in Boston, she has consulted for Janssen and Pfizer and enrolled patients in the studies.

Relkin, who is leading the Gammagard study, said that if all three of these drugs fail, “we’re in trouble.” There hasn’t been a new drug even to help symptoms in nine years, he said.

Petersen of the Mayo Clinic agrees.

“If they’re dead-flat negative, the impact on the field and the implication for Big Pharma could be huge,” he said. Companies “may bail” from the field entirely. “They may just say, ‘This nut is too tough to crack.”’

http://www.timesleader.com/stories/Last-drugs-standing,176933#ixzz20upXXNJ6

 

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