November 10, 2008 by

Opexa to Present Data on its Cellular Therapies for Autoimmune Diseases

The biotech company Opexa announced today that its president and CEO, Neil K. Warma, will deliver a corporate presentation of its patient-specific therapies that are targeted for the treatment of autoimmune diseases such as multiple sclerosis (MS) and diabetes. The presentation will be at Rodman & Renshaw’s 10th Annual Health Care Conference in New York City and will include an overview of the company’s ongoing development program for Tovaxin which is an individualized T-cell therapeutic vaccine that is being developed for the treatment of MS, and which recently yielded positive data from a Phase IIb clinical trial in which 150 patients participated in the multi-center, randomized, double blind, placebo controlled study for the treatment of the Relapsing-Remitting (RR) form of multiple sclerosis. Among other measurements, MRI scans showed statistically significant decreased lesions in those patients who had received Tovaxin.

Tovaxin, which requires only 5 subcutaneous injections per year, is an individualized T-cell therapeutic vaccine based upon attenuated patient-specific myelin-reactive T-cells against peptides of protein from myelin basic protein, myelin oligodendrocyte glycoprotein and proteolipid protein or combinations thereof. Tovaxin is manufactured in Opexa’s in-house cGMP facility.

Tovaxin’s dual mechanism of action combats the demyelination of the nerve fibers in the central nervous system, which is the underlying cause of MS. Clinical results have demonstrated that Tovaxin induces an immune response that depletes and regulates the circulating pathogenic myelin-reactive T-cells that are responsible for attacking the myelin sheath of nerve fibers. Additionally, Tovaxin also rebalances the systemic immune response by causing a shift from pathogenic inflammatory T-cells to anti-inflammatory T-cells.

Opexa Therapeutics is focused on the development and commercialization of patient-specific autologous (in which the donor and recipient are the same person) cellular therapies that are based upon proprietary T-cell and adult stem cell products for the treatment of autoimmune diseases such as multiple sclerosis, rheumatoid arthritis and diabetes. The Company holds the exclusive worldwide license for adult multipotent stem cells derived from mononuclear cells of peripheral blood, which allow large quantities of monocyte-derived stem cells to be produced efficiently for use in autologous therapy, thereby eliminating the risk of immune rejection. In addition to Tovaxin, the T-cell therapy for MS which is currently in Phase IIb clinical trials, Opexa is also in the preclinical development of another product for diabetes mellitus.

Filed Under: Adult Stem Cells, Multiple Sclerosis, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , , , ,
November 5, 2008 by

Genzyme and Osiris Form Adult Stem Cell Mega-Partnership

The biotech companies Osiris Therapeutics and Genzyme announced their signing of a collaboration for the commercialization of two adult stem cell products. According to the agreement, Osiris will commercialize the proprietary products Prochymal and Chondrogen in the U.S. and Canada, while Genzyme will commercialize the products throughout the rest of the world. Genzyme will make an upfront payment of $130 million to Osiris in additon to milestone and royalty payments that are estimated to be worth approximately $1.3 billion. Prochymal and Chondrogen are therapies that use allogeneic (in which the donor and recipient are different people) adult mesenchymal stem cells derived from bone marrow. Both of the products are late-stage treatments for a wide variety of diseases.

Although Osiris developed the therapies, Genzyme will provide the financial support for further marketing and commercialization of the products. This strategic alliance will leverage the leadership position that Osiris enjoys in the development of novel adult stem cell therapies, as well as Genzyme’s regulatory and marketing infrastructure outside of the U.S. and its expertise in the commercialization of cell therapies.

Genzyme and Osiris are already strategic allies and this is not their first collaboration. In 2007, the two companies forged an alliance to develop Prochymal for acute radiation syndrome, which resulted in both companies being awarded a $224.7 million contract in January of 2008 by the U.S. Department of Defense to develop Prochymal for the treatment of radiation-induced medical conditions related to warfare and terrorism. Once Prochymal is approved by the FDA for such indications, the Pentagon will buy 20,000 doses at $10,000 each.

According to Dr. Henri A. Termeer, Genzyme’s chairman and CEO, “This partnership further strengthens Genzyme’s late-stage pipeline of products with the potential to support our growth beyond 2011. Osiris is the clear leader in stem cell technology, which holds the promise to transform standards of care in a number of therapeutic areas in which Genzyme already has a strong presence.”

Similarly, Dr. C. Randal Mills, president and CEO of Osiris, adds, “Today Genzyme and Osiris have forged a powerful partnership in the emerging field of stem cell therapy. This relationship greatly enhances our ability to effectively introduce this groundbreaking technology on a global basis.”

As stated by Edward Tenthoff, an analyst at Piper Jaffray & Co., the deal is the largest ever in the field of stem cell therapy. The stem cells under consideration are exclusively adult stem cells, which are derived from mature tissues instead of embryos. According to Tenthoff, “This is a huge validation. This is a major win for everyone involved.”

Osiris is an adult stem cell company which was founded in 1992 and went public in 2006. Yesterday the company’s stock gained 43 cents, or 2.8%, settling at $15.93 a share after the company announced a third-quarter profit of $5.3 million. Its products focus on the treatment of inflammatory, orthopedic and cardiovascular condtions. The company’s adult stem cell product Prochymal is the only stem cell therapeutic product currently designated by the FDA as both an Orphan Drug and as a Fast Track product. Prochymal is currently being evaluated in three separate phase III clinical trials, two of which are for graft vs. host disease (GvHD) and the third of which is for Chron’s disease, both of which are potentially fatal conditions. Prochymal is also in phase II clinical trials for the regeneration of pancreatic beta islet cells in patients with type I diabetes and for the repair and regeneration of damaged lung tissue in patients with chronic obstructive pulmonary disease. Data from all clinical trials are expected in 2009. Prochymal has also been approved to begin phase II clinical trials as a treatment for the regeneration of cardiac tissue following myocardial infarction. Additionallly, Chondrogen has also been approved to begin phase II and III clinical trials for osteoarthritis of the knee. Osiris currently has 47 U.S. patents, each with one or more foreign counterparts.

Genzyme, the world’s largest maker of drugs for rare genetic disorders, is well known for commercializing first-in-class biotechnologies. Founded in 1981, today Genzyme has a staff of more than 10,000 employees around the globe, with revenues of $3.8 billion. In 2007 Genzyme was awarded the National Medal of Technology, which is the highest honor awarded by the President of the United States for technological innovation. In addition to its large orthopedic franchise, Genzyme’s products are focused on rare genetic disorders, kidney disease, cancer, transplant and immune disease, diagnostic testing, cardiovascular disease, neurodegenrative diseases, endocrinology and other medical specializations in which patient needs are not adequately met. Most recently, Genzyme has also begun developing Mozobil, a novel proprietary product which stimulates the mobilization of the body’s own endogenous stem cells. Genzyme’s latest agreement with Osiris marks a major expansion by Genzyme into the field of cell transplant therapies.

As novel proprietary late-stage adult stem cell treatments which have already been shown to control inflammation, to prevent scarring and to promote tissue regeneration, both Prochymal and Chondrogen have the potential to treat a vast range of diseases. The mutual collaborative development and commercialization of these two adult stem cell therapies, by two of the most prominent biotech industry leaders, signifies an important milestone not only in the histories of these two leading companies, but also in the maturation of the adult stem cell industry. As David Meeker, executive vice president for Genzyme, explains, “The technology has evolved to a point where we have a level of confidence where we’re willing to make the deal. We’ll be working on the clinical development going forward and preparing for commercial launch.”

The partnership involves stem cell therapies which consist exclusively of adult stem cells, not embryonic stem cells, since embryonic stem cells have proven to be highly problematic in the laboratory and have therefore never advanced to the clinical stage.

Filed Under: News, Osteoarthritis, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
October 23, 2008 by

The Potentiation of Mesenchymal Stem Cells Improves Cardiac Regeneration

Mesenchymal stem cells (MSCs) are clinically attractive for a number of reasons which include, among other desirable qualities, their ease of intravenous administration, their ability to home-in on injured tissue, their proven ability to differentiate into a wide variety of tissue types, and their status as immune privileged “universal donor” cells, for which they are especially well known. Numerous clinical trials throughout the U.S. are currently in progress in which MSCs are intravenously administered to patients with a vast range of conditions, thereby validating MSCs as an already well-established and viable therapy. Especially in the treatment of myocardial infarction, allogeneic (in which the donor and recipient are different people) MSCs as a clinical therapy have yielded statistically significant benefits in cardiac patients.

Nevertheless, despite the already high level of success enjoyed by MSCs, scientists have been trying to improve the therapeutic efficacy of these highly potent adult stem cells even further. Now, through a process of molecular potentiation, a team of researchers has succeeded in attaining the goal.

Using MSCs which were modified to overexpress IGF-1, Dr. Husnain Haider and his colleagues at the University of Cincinnati in Ohio observed an overall improvement in cardiac regeneration which was associated with increased mobilization of endogenous bone marrow stem cells in an animal model of heart attack. The MSCs were transfected with this insulin-like growth factor gene, which previously has been found to play an important role in the efficacy of MSCs in a variety of therapeutic uses, including the reversal of kidney failure. Now Dr. Haider’s study indicates that the IGF-1-transfected MSCs also possess superior efficacy in inhibiting pathological changes in rats following myocardial infarction, through the CXCR4 (a CXC chemokine receptor) signaling mechanism in the paracrine release of SDF-1-alpha (stromal-derived-factor being a chemotactically active molecule for lymphocytes), which promoted improved survival and engraftment by the MSCs in the infarcted cardiac tissue. Among other roles, CXCR4, also known as fusin, is specific for SDF-1 (also known as CXCL12) and has been identified in the homing ability of hematopoietic stem cells, and is already recognized as an important receptor in a wide variety of molecular processes.

Dr. Haider’s results corroborate a recent study conducted by Dr. Kondo of Japan, in which angiogenesis from exogenously administered bone marrow stem cells in an animal model of critical limb ischemia was found to be highly dependent upon the moblization of endogenous bone marrow stem cells which were activated by the exogenous stem cells. Now Dr. Haider’s group has observed that the ability of the exogenously administered MSCs to repair cardiac tissue and to inhibit further post-infarct pathological changes following a heart attack is also dependent upon the mobilization of endogenous bone marrow stem cells.

The role of IGF-1 in mobilizing endogenous bone marrow stem cells through paracrine activation of SDF-1-alpha/CXCR4 signaling, thereby increasing and improving the therapeutic efficacy of MSCs, is a discovery which may also prove to have additional applications in the potentiation of other types of stem cells.

Filed Under: Heart Failure, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
October 19, 2008 by

Australian Scientists Derive Heart Muscle From Fat Cells

Adult stem cells that have been derived from human adipose (fat) tissue mark the first of their kind in a new breakthrough that could offer an ideal stem cell therapy for heart patients.

According to Dr. Rodney Dilley, principal scientist at Melbourne’s Bernard O’Brien Institute, “The fact that you can do this potentially opens a whole area of heart regeneration methods. Our approach is to create a piece of heart muscle that we can use to put onto the heart to stop it from remodelling and to return its contractile function to normal.” Since heart muscle does not usually regenerate itself after injury, but instead forms scar tissue as part of the “remodelling” process, this announcement by the Australian scientists has far-reaching implications for the field of cardiology. Additionally, since most people have accumulated the storage of some body fat, adipose tissue constitutes one of the most easily accessible sources of autologous adult stem cells.

The discovery could potentially offer a treatment for a wide variety of cardiac problems, ranging from congenital heart defects to age-related heart disease.

Filed Under: Heart Disease, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
October 15, 2008 by

Neuralstem Licenses Technology for Spinal Cord Treatment

Neuralstem has licensed the patent rights for three inventions from the Cleveland Clinic, all of which utilize spinal cord injection technology, and which Neuralstem will use in clinical trials for ALS (Amyotrophic Lateral Sclerosis, also known as Lou Gehrig’s disease). All three inventions were developed by Dr. Nicholas Boulis, currently at Emory University but formerly of the Cleveland Clinic. Neuralstem plans to file its IND (Investigational New Drug) application with the FDA before the end of this year, with trials scheduled to commence in early 2009.

According to Neuralstem President and CEO, Dr. Richard Garr, “We are very pleased to have licensed-in these important delivery technology rights. As we get ready to move into our expected human trial for ALS, we are focused on the optimal way to deliver our cell therapeutics to enhance the safety and efficacy of the treatments. Transplanting our cells directly into the spinal cord is a challenging but necessary part of trying to treat diseases of the spinal cord, not only in ALS but in other spinal cord injuries and diseases. The technology today will help us meet those goals.”

Specifically, the three inventions consist of devices which are known as a “stabilized platform and microelectrode recording guidance validation”, a “floating cannula for spinal cord therapeutic injection”, and a “spinal platform and methods for delivering a therapeutic agent to a spinal cord target”. According to its website, “Neuralstem, Inc. is a biotherapeutics company utilizing its patented Human Neural Stem Cell technology to create cures for diseases of the CNS (central nervous sysem). The Company’s technology allows for the isolation of CNS stem cells from tissue, the expansion in vitro of each cell up to a billion-billion times (60 doublings), and the controlled differentiation of the cells into mature, physiologically relevant human neurons and glia.”

In addition to ALS, ischemic paraplegia, traumatic spinal cord injury, and Parkinson’s disease are also among Neuralstem’s primary areas of focus.

Filed Under: News, Spinal Cord Injury, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
October 1, 2008 by

Pfizer Expands Into Stem Cell Research

As a medical therapy, stem cells offer, for the first time in history, the possibility of treatment and perhaps even the cure of human diseases which previously have been untreatable. Precisely for that reason, the business of stem cells is projected to be a lucrative one.

The stem cell field is estimated to become a $500 billion industry over the next 20 years, and there is hardly a nation on earth that is not targeting stem cell research and development as part of its economy. To be able to “get in at the ground floor”, in any business with this potential for growth, is a rare opportunity. Recognizing such an obvious fact, the largest pharmaceutical company in the world has now decided to seize this opportunity.

According to Dr. John McNeish, executive director of R&D at Pfizer, the pharmaceutical industry leader is scheduled to open its second regenerative medicine center in Cambridge, England, next month. The focus of its U.K. location will be iPS (induced pluripotent stem) cells and their applications in ophthalmologic and central nervous system diseases. Pfizer’s first regenerative medicine center, located in Cambridge, Massachusetts, already focuses on stem cell therapies for the treatment of heart disease and diabetes.

As Dr. McNeish announced to reporters last month at the World Stem Cell Summit that was held in Madison, Wisconsin, “Stem cells can help us make good decisions about which compounds will be more likely to be safe. These cells will be tremendous in drug discovery. They will help us understand personalized medicine, genetic variation, ethnic populations, and which biomarkers to follow.”

As the largest pharmaceutical company in the world, Pfizer employs approximately 100,000 people worldwide in the manufacture and commercialization of prescription medication, with sales of Lipitor, its cholesterol-lowering drug, exceeding $10 billion last year alone. As an indication of its commitment to the stem cell field, Pfizer’s new regenerative medicine center in Cambridge, England, is estimated to occupy a space of approximately 52,000 square feet in area.

Pfizer is not the first pharmaceutical company to enter the stem cell industry. Last year, GlaxoSmithKline (GSK), AstraZeneca and Roche Holding together launched a new drug screening initiative entitled “Stem Cells for Safer Medicines”. GSK has also announced a $25 million collaboration with the Harvard Stem Cell Institute, geared toward developing the drug screening potential of stem cell technology.

The merging of “big pharma” with stem cell R&D is perhaps the latest and most significant indication of the rapid growth of the stem cell field, though this will certainly not be the last indication of its type.

Filed Under: Heart Disease, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
August 11, 2008 by

Pluristem’s Adult Stem Cell Product Shows Promise as Therapy for Multiple Sclerosis

Pluristem Therapeutics, Inc., which specializes in the commercialization of adult stem cell therapy products for the treatment of degenerative, ischemic and autoimmune diseases, has demonstrated success in an animal model with a product that it has developed specifically for the treatment of multiple sclerosis (MS). Known as PLX-MS, for “PLacental eXpanded” cells, this product, which is derived entirely from adult stem cells, has proven to be effective in vivo. Specifically, mice that received PLX-MS did not develop clinical symptoms associated with MS when compared with control mice, for the entirety of the 35-day study. PLX cells are mesenchymal stromal cells that are derived from placental blood and then expanded under Pluristem’s proprietary technology.

According to Zami Aberman, president and CEO of Pluristem, “This trial’s remarkable results demonstrated our PLX-MS cells’ ability to prevent the appearance of multiple sclerosis symptoms and showed the potential for our PLX cells to treat global autoimmune diseases. As a cellular therapy, our PLX cells, which are derived from human placenta, a non-controversial, non-embryonic, adult stem cell source, and stored ready-to-use, could prove to be a readily available preventive therapeutic alternative for these disorders.”

The World Health Organization (WHO) estimates that approximately 2.5 million people throughout the world suffer from multiple sclerosis, which is an autoimmune, demyelinating disease of the central nervous system for which there has previously been no cure, and only limited therapies available prior to the advent of stem cell technology. At its current level, the global market for MS treatments has been estimated at approximately $5.4 billion per year, although this figure is expected to rise based upon epidemiological projections.

Filed Under: Adult Stem Cells, Multiple Sclerosis, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , , , ,
July 14, 2008 by

Two Leading Biotech Companies Collaborate on a Department of Defense Contract for Adult Stem Cell Applications in Warfare

In January of 2008, Osiris Therapeutics and Genzyme began collaborating together on a multi-million-dollar Department of Defense (DoD) contract that was awarded to Osiris, the objective of which is the development of a civilian and military medical response to nuclear or radiological events.

The DoD contract specifies the development and stockpiling of Prochymal, which is a proprietary adult stem cell therapy developed by Osiris, specifically for the repair of cellular injury that might result from the “acute radiation syndrome” (ARS) that accompanies severe and sudden radiation exposure. Terms of the contract provide for the purchase of up to 20,000 doses of Prochymal by the DoD at $10,000 per dose.

According to C. Randal Mills, Ph.D., President and CEO of Osiris, “We are honored that the Department of Defense has selected Prochymal in this critical effort to better safeguard our armed forces against the potentially horrendous effects of battlefield exposure to a radiological weapon. The contract also brings into focus a substantial new market opportunity for Prochymal. We are working diligently towards licensure of Prochymal for ARS and stand ready to assist other sectors of the United States government and allied nations in their emergency preparedness efforts.”

Major General John Parker, M.D., a former Commanding General who is currently responsible for countermeasure development and acquisition and who is also a member of the Medical Countermeasure Advisory Board of Osiris, adds, “Prochymal’s unique mechanism of action and strong clinical profile make it very well suited to address the complicated injuries associated with ARS. Currently, every scenario contemplating a radiological emergency, both civilian and military, involves people suffering from the life-threatening effects of ARS without effective treatments. Today’s decision by DoD sets in motion a sound plan to change that, by expeditiously completing development of the first effective therapy for ARS.”

As Henri Termeer, Chairman and CEO of Genzyme, explains, “We are pleased to partner with Osiris in developing this innovative cell therapy to treat the potentially lethal complications of ARS for the U.S. military. With our combined first-in-class technology and development expertise, Osiris and Genzyme have the necessary resources to complete this assignment for the Department of Defense and to work with other government organizations committed to safeguarding our nation and its allies.” According to Thomas MacVittie, Ph.D., Professor of Radiation Oncology and Pathology at the University of Maryland and a member of the NIAID (National Institute of Allergy and Infectious Diseases) Medical Countermeasures and CDC Strategic National Stockpile Radiation Working Groups, who is also a member of the Medical Countermeasure Advisory Board of Osiris, “Prochymal represents a breakthrough in countermeasure development for ARS. Prochymal has demonstrated therapeutic utility in humans repairing many of the major organ systems affected by radiation injury. Where most approaches only target a single component of ARS, Prochymal has the potential to address the entire syndrome including both acute and delayed effects in multiple organ systems.”

ARS is known to damage most severely the DNA of the rapidly dividing cells in the gastrointestinal tract, the skin and bone marrow. If severe and untreated, death can result within a matter of days or months following the initial exposure. Prochymal is a highly purified formulation of mesenchymal stem cells that are cultured and expanded. Prochymal is currently in Phase II clinical trials for the treatment of Type I diabetes, and Phase III clinical trials for both the treatment of Graft vs. Host Disease and Crohn’s disease. Additionally, Prochymal has demonstrated preliminary efficacy in the treatment of heart attacks and it has demonstrated a strong safety profile in seven previous Phase I and Phase II clinical trials. Prochymal has also shown a potential ability to reverse cellular damage and improve survival in diseases that are similar to ARS.

While warfare has always been understood to have biological and health consequences for those who are involved, the anti-inflammatory and regenerative properties of adult stem cells now offer a new type of countermeasure against nuclear and radiological threats. The strong interest of the Deparment of Defense in adult stem cell therapies marks yet another historic milestone in the versatility and applicability of these potent therapies.

Filed Under: Heart Disease, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
July 14, 2008 by

FDA Authorizes Device for Bone Marrow Stem Cell Preparation

The ThermoGenesis Corporation, which manufactures automated blood processing systems and disposable products that are used in the manufacture, preservation and delivery of cell and tissue therapy products, including the processing and storage of adult stem cells, announced today that it has received FDA authorization to begin marketing its MarrowXpress (MXP) device. The MXP is used for the preparation of the adult stem cell concentrate that is derived from bone marrow, and as such the device is applicable both in intraoperative and clinical laboratory settings.

In June, ThermoGenesis submitted a pre-market notification application to the FDA, which subsequently determined that the MXP is exempt from pre-market notification requirements and instead will be subject to regulation that governs laboratory equipment which is designated for specific medical utilization. According to Dr. William Osgood, CEO of ThermoGenesis, “This notification that we can immediately begin marketing our MXP device is a major regulatory milestone for the company and particularly exciting since we received this notification just several weeks after filing our submission, and since it follows by less than a month from having received the CE-Mark enabling us to market the device in the European Community. In anticipation of this authorization, we have initiated discussions with an orthopedic surgery group, vascular surgeons, and leading academic medical centers not only in the U.S. but also in Europe and Asia, and we look forward to begin placing this device in the clinical setting in the near future. We believe this positions ThermoGenesis to significantly participate in what will ultimatel represent a multi-billion dollar market opportunity in regenerative medicine.”

Bone marrow is the leading source for adult stem cells that are currently in clinical trials for the treatment of a variety of diseases which include, most predominantly, ischemic heart disease, peripheral artery disease, diabetes and blood disorders. According to the U.S. Centers for Disease Control and Prevention, approximately 24 million people in the U.S. alone are known to have heart disease, while approximately 16.2 million people have peripheral artery disease and another 15.8 million people suffer with diabetes. Such statistics for the U.S. comprise nearly half of all documented cases of these diseases worldwide. While the MXP is specifically designed for the processing of stem cells from bone marrow, similar technologies are also being developed with applications to adult stem cells that are derivable from blood and fat.

According to Dr. John Chapman, Vice President of Research and Development and Scientific Affiars at ThermoGenesis, “We are confident that the users of this technology will value the unique capability of this device to achieve significantly improved recovery of the stem cells from bone marrow, meaning that less marrow will need to be collected from their patients to obtain the same number of stem cells. While the initial marketing application for the MXP will be for bone marrow processing, both at point-of-care and in a laboratory, we will continue our exploration of the use of this technology platform for other sources of adult stem cells including mobilized peripheral blood and adipose tissue stem cell processing.”

This latest FDA authorization of a newly patented medical device represents merely one more example of the explosive growth that is occurring in the regenerative medicine industry, not only in regard to the biological materials themselves, such as the stem cells, that are used, but also in regard to the electronic machinery and engineering that are helping to drive the medical advancements, and for which there is also an increasing global market.

Filed Under: Heart Disease, News, Stem Cell Research, Stem Cells, Uncategorized Tagged With: , , , ,
July 11, 2008 by

Large-Scale Adult Stem Cell Trials Planned for Children With Sickle Cell Anemia

In an article published in the August 2007 issue of the Journal of Bone Marrow Transplantation, Dr. Shalini Shenoy reviewed the latest advancements in stem cell transplantation which allow for low toxicity and high success rates in the treatment of sickle cell anemia. However, as Dr. Shenoy pointed out, stem cell therapies are not yet in common use for the treatment of this disease. Today, Dr. Shenoy is now leading one of the largest studies ever to be conducted in the treatment of sickle cell anemia, as this comprehensive clinical trial is designed specifically to test the safety and efficacy of stem cell therapy on a grand scale. As associate professor of pediatrics at the Washington University School of Medicine, and medical director of the pediatric bone marrow transplant program at St. Louis Children’s Hospital, Dr. Shenoy is directing the nationwide, multicenter Phase II clinical trials for the use of adult stem cell therapy in the treatment of children with the most advanced forms of the disease. Qualifying participants will receive stem cells derived either from bone marrow or umbilical cord blood. A total of 45 patients are sought, between the ages of 3 and 16, who suffer from the most severe, most life-threatening forms of sickle cell anemia.

According to Dr. Shenoy, “Right now, blood stem cell transplant is the only potential curative therapy for severe sickle cell disease.” Approximately ten patients with sickle cell anemia improved after receiving stem cell transplants in preliminary trials, the results of which were so positive that interest was generated in expanding the trials to a larger population group.

An inherited blood disorder in which hemoglobin is abnormally shaped, sickle cell anemia afflicts approximately 70,000 people in the U.S. alone, occurring in 1 in every 500 African-American births, and 1 in approximately every 1,200 Hispanic-American births. Previously, blood transfusions and bone marrow transplants have offered the only known treatments, both of which have serious complications which include graft-versus-host disease and a 10% mortality rate from bone marrow transplantation. By contrast, hematopoietic stem cell therapy has been shown to restore normal hematopoiesis to people who are suffering with sickle cell anemia, with very low risks.

The upcoming nationwide trial is supported by the National Marrow Donor Program, the Sickle Cell Disease Clinical Research Network, the Bone Marrow Transplant Clinical Trials Network of the National Heart, Lung and Blood Institute, and the Pediatric Blood and Marrow Transplant Consortium.

Filed Under: Heart Disease, News, Stem Cells, Uncategorized Tagged With: , , , ,
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