Category Archives: Stem Cell Therapy

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'

4.5 (2 votes)

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

Editor's Choice Main Category: Pediatrics / Children's Health Also Included In: Stem Cell Research Article Date: 20 May 2012 - 11:00 PDT

Current ratings for: 'World's First Stem Cell Drug From Osiris : Approved!'

5 (1 votes)

The decision is a historic one, as it's both the first stem cell drug going into formal use, as well as the first treatment for GvHD. The disease is a devastating breakdown occurring after a bone marrow transplant and kills around 80% of children affected, often within a matter of weeks.

Andrew Daly, M.D., Clinical Associate Professor, Department of Medicine and Oncology at the University of Calgary, Canada and Principal Investigator in the phase 3 clinical program for Prochymal confirmed :

The approval process for Prochymal was implemented under Health Canada's Notice of Compliance with conditions (NOC/c) pathway. The basis of the procedure allows a new drug to come onto the market where there are unmet medical needs. The approval is granted with the provision that the drug has demonstrated risk / reward benefits in previous clinical trials and that the manufacturer agrees to undertake additional confirmatory clinical testing.

C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris confirmed his' companies happiness at being able to help conquer the disease :

Where children with GvHD are not responding to treatment with steroids, which is presumably most of them, the use of Prochymal will now be authorized. Health Canada based it's approval on previous clinical studies of the drug, in which 64% of patients showed results; the survival rate compared to historical data was drastically improved, even in patients with severe cases. Additional clinical evaluation of Prochymal now will be undertaken, including enrolling patients in a registry to discover any long term effects.

Joanne Kurtzberg, MD, Head of the Pediatric Bone Marrow Transplant Program at Duke University and Lead Investigator for Prochymal

Osiris has 48 patents protecting Prochymal, and Health Canada's have agreed to provide Prochymal with regulatory exclusivity within their territory. Canada affords eight years of exclusivity to Innovative Drugs, such as Prochymal, with an additional six-month extension because it addresses a pediatric disease. Parents, doctors and shareholders can all rest easy.

Excerpt from:
World's First Stem Cell Drug From Osiris : Approved!

Indian clinic's stem cell therapy real?

STORY HIGHLIGHTS

For more of CNN correspondent Drew Griffin's investigation of India's experimental embryonic stem cell therapy, watch "CNN Presents: Selling a Miracle," at 8 and 11 p.m. ET Sunday on CNN.

New Delhi (CNN) -- Cash Burnaman, a 6-year-old South Carolina boy, has traveled with his parents to India seeking treatment for a rare genetic condition that has left him developmentally disabled. You might think this was a hopeful mission until you learn that an overwhelming number of medical experts insist the treatment will have zero effect.

Cash is mute. He walks with the aid of braces. To battle his incurable condition, which is so rare it doesn't have a name, Cash has had to take an artificial growth hormone for most of his life.

His divorced parents, Josh Burnaman and Stephanie Krolick, are so driven by their hope and desperation to help Cash they've journeyed to the other side of the globe and paid tens of thousands of dollars to have Cash undergo experimental injections of human embryonic stem cells.

The family is among a growing number of Americans seeking the treatment in India -- some at a clinic in the heart of New Delhi called NuTech Mediworld run by Dr. Geeta Shroff, a retired obstetrician and self-taught embryonic stem cell practitioner.

Shroff first treated Cash -- who presents symptoms similar to Down Syndrome -- in 2010. "I am helping improve their quality of life," Shroff told CNN.

After five weeks of treatment, Cash and his parents returned home to the U.S.

That's when Cash began walking with the aid of braces for the first time.

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Medical success or boondoggle?