Editor's Choice Main Category: Bones / Orthopedics Also Included In: Stem Cell Research Article Date: 22 May 2012 - 12:00 PDT
Current ratings for: 'Prochymal - First Stem Cell Drug Approved'
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Prochymal (remestemcel-L) is also the first drug to be approved for the treatment of acute graft-vs-host disease (GvHD) in children, a devastating complication of bone marrow transplantation that kills almost 80% of all affected children, many of which just weeks after they have been diagnosed.
GvHD is the leading cause of transplant-related mortality, caused by an immunologic attack. Severe GvHD can cause blistering of the skin, intestinal hemorrhage and liver failure and is extremely painful with a death rate of up to 80%. At present, the first-line standard therapies for GvHD are steroids. Given that the success rate of steroids is only 30 to 50%, the only other therapy if steroids fail is limited to immunosuppressive agents that are used off-label with little benefit and significant toxicities. Until the approval of Prochymal, there has not been any other therapy for GvHD.
Osiris Therapeutics Inc. was awarded authorization for Prochymal under Health Canada's Notice of Compliance with conditions (NOC/c). A NOC/c is an authorization to market a drug with the condition that the manufacturer undertakes additional studies to verify the clinical benefit. This pathway provides access to treatments for unmet medical conditions and has demonstrated its benefits outweigh its risks in clinical trials.
Andrew Daly, M.D., Clinical Associate Professor from the Department of Medicine and Oncology at the University of Calgary, Canada and leading researcher of Prochymal's phase 3 clinical program declared:
C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris announced:
Prochymal is an intravenous formulation of mesenchymal stem cells (MSCs), which are derived from the bone marrow of healthy adult donors aged between 18 and 30 years. The MSCs are selected from the bone marrow and grown in culture, producing up to 10,000 doses of Prochymal from a single donor. The drug is a true off-the-shelf stem cell product, which is stored frozen until it is needed. Prochymal is infused through a simple intravenous line without the need to type or immunosuppress the recipient. The drug is currently undergoing Phase 3 trials for refractory Crohn's disease, as well as undergoing clinical trials for the treatment of heart attacks and type-1 diabetes.
Health Canada granted the Prochymal's authorization for the management of acute GvHD in children who are unresponsive to steroids, based on the drug's results of clinical trials. Prochymal was recommended by an independent expert advisory panel, which was commissioned to assess the drugs safety and efficacy.
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Prochymal - First Stem Cell Drug Approved