Category Archives: Stem Cells

CAMBRIDGE, Mass.--(BUSINESS WIRE)--

Verastem, Inc., (VSTM) a clinical-stage biopharmaceutical company focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, announced the appointment of Alison Lawton and Michael Kauffman, M.D., Ph.D., to its Board of Directors.

Together, Ms. Lawton and Dr. Kauffman bring leadership in the clinical, regulatory and commercial aspects of the biopharmaceutical industry to Verastem at a transformative time in our development, said Henri Termeer, Lead Director of Verastem. On behalf of the Verastem Board of Directors, I am very pleased to welcome Alison and Michael and look forward to their contributions as we begin to demonstrate the clinical benefits of targeting cancer stem cells.

In conjunction with the appointments, Steven Kraus of Bessemer Venture Partners and Ansbert Gadicke, M.D., of MPM Capital have stepped down from the Board of Directors.

We deeply thank Steve and Ansbert for their valuable service to Verastem, said Christoph Westphal, M.D., Ph.D., Chairman and CEO of Verastem. They have made important contributions as we progressed from translating the pioneering research on cancer stem cells by Dr. Bob Weinberg to the design of clinical trials, including a potential registration trial of VS-6063 next year.

Alison Lawton has been with Genzyme Corp. (now NYSE: SNY) for 21 years and is currently Senior Vice President and General Manager of the Sanofi Biosurgery Business Unit. Ms. Lawton has led global functional organizations including Regulatory Affairs and Corporate Quality Systems, Policy Programs, Health Outcomes and Strategic Pricing, Patient Safety and Risk Management and BMRA Process Excellence and Training. Ms. Lawton spent 8 years in the UK at Parke-Davis and is past President and Chair of the Board of Regulatory Affairs Professional Society and is currently a director of Cubist Pharmaceuticals (CBST) and MassMEDIC.

Verastem has a unique insight into cancer biology, remarkable scientific leadership and a highly dedicated management team focused on eradicating a cause of cancer recurrence and metastasis, said Ms. Lawton. I am excited to contribute to Verastem as the Company plans to initiate important clinical trials.

Dr. Michael Kauffman is the cofounding CEO of Karyopharm Therapeutics. Previously, he was the Chief Medical Officer of Onyx Pharmaceuticals Inc., where he was a key leader in the development of Kyprolis (carfilzomib), a novel proteasome inhibitor approved in refractory myeloma. Dr. Kauffman was CEO of Proteolix (now Onyx) and is past President and Chief Executive Officer of EPIX Pharmaceuticals, Inc. (previously Predix Pharmaceuticals, Inc.). He played a key role in the Velcade Development Program at Millennium Pharmaceuticals, and held a number of senior positions at Millennium Predictive Medicine and Biogen. Dr. Kauffman received his MD and Ph.D. from Johns Hopkins Medical School and is board certified in internal medicine.

I believe that Verastem is on the leading edge of realizing the immense therapeutic potential of targeting cancer stem cells, said Dr. Kauffman. I am particularly enthusiastic about the identification of biomarkers to aid efficient trial design and identification of patients most likely to respond to treatment.

AboutVerastem, Inc.

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Alison Lawton and Michael Kauffman Join Verastem Board of Directors

Public release date: 26-Nov-2012 [ | E-mail | Share ]

Contact: Shawna Williams shawna@jhmi.edu 410-955-8236 Johns Hopkins Medicine

Johns Hopkins researchers report concrete steps in the use of human stem cells to test how diseased cells respond to drugs. Their success highlights a pathway toward faster, cheaper drug development for some genetic illnesses, as well as the ability to pre-test a therapy's safety and effectiveness on cultured clones of a patient's own cells.

The project, described in an article published November 25 on the website of the journal Nature Biotechnology, began several years ago, when Gabsang Lee, D.V.M., Ph.D., an assistant professor at the Johns Hopkins University School of Medicine's Institute for Cell Engineering, was a postdoctoral fellow at Sloan-Kettering Institute in New York. To see if induced pluripotent stem cells (iPSCs) could be used to make specialized disease cells for quick and easy drug testing, Lee and his colleagues extracted cells from the skin of a person with a rare genetic disease called Riley-Day syndrome, chosen because it affects only one type of nerve cell that is difficult if not impossible to extract directly from a traditional biopsy. These traits made Riley-Day an ideal candidate for alternative ways of generating cells for study.

In a so-called "proof of concept" experiment, the researchers biochemically reprogrammed the skin cells from the patient to form iPSCs, which can grow into any cell type in the body. The team then induced the iPSCs to grow into nerve cells. "Because we could study the nerve cells directly, we could for the first time see exactly what was going wrong in this disease," says Lee. Some symptoms of Riley-Day syndrome are insensitivity to pain, episodes of vomiting, poor coordination and seizures; only about half of affected patients reach age 30.

In the recent research at Johns Hopkins and Memorial Sloan-Kettering, Lee and his co-workers used these same lab-grown Riley-Day nerve cells to screen about 7,000 drugs for their effects on the diseased cells. With the aid of a robot programmed to analyze the effects, the researchers quickly identified eight compounds for further testing, of which one SKF-86466 ultimately showed promise for stopping or reversing the disease process at the cellular level.

Lee says a clinical trial with SKF-86466 might not be feasible because of the small number of Riley-Day patients worldwide, but suggests that a closely related version of the compound, one that has already been approved by the U.S. Food and Drug Administration for another use, could be employed for the patients after a few tests.

The implications of the experiment reach beyond Riley-Day syndrome, however. "There are many rare, 'orphan' genetic diseases that will never be addressed through the costly current model of drug development," Lee explains. "We've shown that there may be another way forward to treat these illnesses."

Another application of the new stem cell process could be treatments tailored not only to an illness, but also to an individual patient, Lee says. That is, iPSCs could be made for a patient, then used to create a laboratory culture of, for example, pancreatic cells, in the case of a patient with type 1 diabetes. The efficacy and safety of various drugs could then be tested on the cultured cells, and doctors could use the results to help determine the best treatment. "This approach could move much of the trial-and-error process of beginning a new treatment from the patient to the petri dish, and help people to get better faster," says Lee.

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Study advances use of stem cells in personalized medicine

ScienceDaily (Nov. 26, 2012) Johns Hopkins researchers report concrete steps in the use of human stem cells to test how diseased cells respond to drugs. Their success highlights a pathway toward faster, cheaper drug development for some genetic illnesses, as well as the ability to pre-test a therapy's safety and effectiveness on cultured clones of a patient's own cells.

The project, described in an article published November 25 on the website of the journal Nature Biotechnology, began several years ago, when Gabsang Lee, D.V.M., Ph.D., an assistant professor at the Johns Hopkins University School of Medicine's Institute for Cell Engineering, was a postdoctoral fellow at Sloan-Kettering Institute in New York. To see if induced pluripotent stem cells (iPSCs) could be used to make specialized disease cells for quick and easy drug testing, Lee and his colleagues extracted cells from the skin of a person with a rare genetic disease called Riley-Day syndrome, chosen because it affects only one type of nerve cell that is difficult if not impossible to extract directly from a traditional biopsy. These traits made Riley-Day an ideal candidate for alternative ways of generating cells for study.

In a so-called "proof of concept" experiment, the researchers biochemically reprogrammed the skin cells from the patient to form iPSCs, which can grow into any cell type in the body. The team then induced the iPSCs to grow into nerve cells. "Because we could study the nerve cells directly, we could for the first time see exactly what was going wrong in this disease," says Lee. Some symptoms of Riley-Day syndrome are insensitivity to pain, episodes of vomiting, poor coordination and seizures; only about half of affected patients reach age 30.

In the recent research at Johns Hopkins and Memorial Sloan-Kettering, Lee and his co-workers used these same lab-grown Riley-Day nerve cells to screen about 7,000 drugs for their effects on the diseased cells. With the aid of a robot programmed to analyze the effects, the researchers quickly identified eight compounds for further testing, of which one -- SKF-86466 -- ultimately showed promise for stopping or reversing the disease process at the cellular level.

Lee says a clinical trial with SKF-86466 might not be feasible because of the small number of Riley-Day patients worldwide, but suggests that a closely related version of the compound, one that has already been approved by the U.S. Food and Drug Administration for another use, could be employed for the patients after a few tests.

The implications of the experiment reach beyond Riley-Day syndrome, however. "There are many rare, 'orphan' genetic diseases that will never be addressed through the costly current model of drug development," Lee explains. "We've shown that there may be another way forward to treat these illnesses."

Another application of the new stem cell process could be treatments tailored not only to an illness, but also to an individual patient, Lee says. That is, iPSCs could be made for a patient, then used to create a laboratory culture of, for example, pancreatic cells, in the case of a patient with type 1 diabetes. The efficacy and safety of various drugs could then be tested on the cultured cells, and doctors could use the results to help determine the best treatment. "This approach could move much of the trial-and-error process of beginning a new treatment from the patient to the petri dish, and help people to get better faster," says Lee.

Other authors of the paper are Christina N. Ramirez, Ph.D., Nadja Zeltner, Ph.D., Becky Liu, Constantin Radu, M.S., Bhavneet Bhinder, Hakim Djaballah, Ph.D., and Lorenz Studer, Ph.D., of the Sloan-Kettering Institute; and Hyesoo Kim, Ph.D., Young Jun Kim, M.D., Ph.D., InYoung Choi, Ph.D., and Bipasha Mukherjee-Clavin of the Johns Hopkins University School of Medicine.

The work was supported by funds from New York State Stem Cell Science (NYSTEM), the New York Stem Cell Foundation (NYSCF), the state of Maryland (TEDCO, MSCRF), the Commonwealth Foundation for Cancer Research, the Experimental Therapeutics Center at Memorial Sloan-Kettering Cancer Center, the William Randolph Hearst Fund in Experimental Therapeutics, the L.S. Wells Foundation, and the National Cancer Institute (grant number 5 P30 CA008748-44).

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Use of stem cells in personalized medicine

ScienceDaily (Nov. 27, 2012) A new method of growing cardiac tissue is teaching old stem cells new tricks. The discovery, which transforms aged stem cells into cells that function like much younger ones, may one day enable scientists to grow cardiac patches for damaged or diseased hearts from a patient's own stem cells -- no matter what age the patient -- while avoiding the threat of rejection.

Stem cell therapies involving donated bone marrow stem cells run the risk of patient rejection in a portion of the population, argues Milica Radisic, Canada Research Chair in Functional Cardiovascular Tissue Engineering at the Institute of Biomaterials and Biomedical Engineering (IBBME) and Associate Professor in the Department of Chemical Engineering and Applied Chemistry at the University of Toronto.

One method of avoiding the risk of rejection has been to use cells derived from a patient's own body. But until now, clinical trials of this kind of therapy using elderly patients' own cells have not been a viable option, since aged cells tend not to function as well as cells from young patients.

"If you want to treat these people with their own cells, how do you do this?"

It's a problem that Radisic and her co-researcher, Dr. Ren-Ke Li, think they might have an answer for: by creating the conditions for a 'fountain of youth' reaction within a tissue culture.

Li holds the Canada Research Chair in Cardiac Regeneration and is a Professor in the Division of Cardiovascular Surgery, cross-appointed to IBBME. He is also a Senior Scientist at the Toronto General Research Institute.

Radisic and Li first create a "micro-environment" that allows heart tissue to grow, with stem cells donated from elderly patients at the Toronto General Hospital.

The cell cultures are then infused with a combination of growth factors -- common factors that cause blood vessel growth and cell proliferation -- positioned in such a way within the porous scaffolding that the cells are able to be stimulated by these factors.

Dr. Li and his team then tracked the molecular changes in the tissue patch cells. "We saw certain aging factors turned off," states Li, citing the levels of two molecules in particular, p16 and RGN, which effectively turned back the clock in the cells, returning them to robust and healthy states.

"It's very exciting research," says Radisic, who was named one of the top innovators under 35 by MIT in 2008 and winner of the 2012 Young Engineers Canada award.

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'Fountain of youth' technique rejuvenates aging stem cells

Dr Madhuravally Thampi on Stem Cells (gynaecology) in Asianet Suprabhatam (Part 1)
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