Canada approves historic life-saving stem-cell drug for children

Prochymal is truly an off-the-shelf stem cell product that is stored frozen until needed and infused through a simple intravenous line.

Canada has become the first country to greenlight the world’s first approved drug that has stem cells as its active ingredient, offering hope to children suffering from an extremely painful disease that kills up to 80 percent of children affected.

“I am very proud of the leadership role Canada has taken in advancing stem cell therapy and particularly gratified that this historic decision benefits children who would otherwise have little hope,” said Andrew Daly, M.D., Clinical Associate Professor, Department of Medicine and Oncology at the University of Calgary, Canada, according to a press release.As the National Institutes of Health explains, the immune system, as the body’s tool to fight infection and disease, works by seeing harmful cells as “foreign” and attacking them.When children receive a donor’s stem cells (the “graft”), their job is to recreate the donor’s immune system in the child’s body (the “host”).The New York Times says, graft versus host disease (GvHD), the leading cause of transplant related deaths, is the term used when immune cells contained within transplanted bone marrow see the recipient’s organs as foreign and attack them, causing potentially severe damage to the skin, liver and digestive tract.The National Institutes of Health says symptoms can include:

Skin GvHD■ red rash■ hair loss■ blistering of the skinLiver GvHD■ elevated liver tests determined through blood tests■ yellowing of the skin and whites of the eyes■ abdominal pain (later symptom)Gastrointestinal (GI) GvHD■ watery diarrhea■ stomach cramps (especially before and during bowel movements and after eating)■ persistent nausea

How is GVHD treated?Doctors try using steroids to stop the attack, but they only have a success rate of 30-50 percent, which is why many children can die within just weeks of diagnosis, the press release states.In Canada, Prochymal is now authorized for the management of acute GvHD in children who fail to respond to steroids.Prochymal, developed by Osiris Therapeutics, is an intravenous formulation of mesenchymal stem cells (MSCs), which are derived from the bone marrow of healthy adult donors between the ages of 18 and 30 years.The MSCs are selected from the bone marrow and grown in culture so that up to 10,000 doses of Prochymal can be produced from a single donor.Because these are adult stem cells, they do not raise the ethical concerns of embryonic stem cells, whose creation usually involves the destruction of human embryos, Reuters adds.As the New York Times notes, stem cells are already used in medicine. Bone marrow or stem cell transplants are used to treat various cancers and genetic diseases. But those transplants are medical procedures, not products sold by a drug company.”As a result of Health Canada’s comprehensive review, physicians now have an off-the-shelf stem cell therapy in their arsenal to fight GvHD. Much like the introduction of antibiotics in the late 1920’s, with stem cells we have now officially taken the first step into this new paradigm of medicine,” Daly said, Principal Investigator in the clinical program for Prochymal.“Today is not only a great day for Osiris, but for everyone involved in the responsible development of stem cell therapies,” said C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris. “Most importantly, today is a great day for children and their families who bravely face this horrific disease. While today marks the first approval of a stem cell drug, now that the door has been opened, it will surely not be the last.”Mills said Thursday that the Food and Drug Administration indicated that it would require more data before approval, prompting Osiris to seek approval in Canada first, according to the Times. He said the company would apply to the F.D.A. later this year.

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