First approval for a stem cell based drug

The first regulatory approval for a manufactured drug based on stem cells has been received for a drug to support bone marrow transplants.

Canadian regulators have approved Prochymal from Osiris Therapeutics to treat children suffering from the rare graft-versus-host disease.

The drug is prepared from stem cells obtained from the bone marrow of healthy young adult donors and replicated in culture.

A clinical trial showed the drug was effective in 60% of children suffering from the condition in whom steroid treatment had failed.

Graft-versus-host disease following bone marrow transplant is a form of transplant rejection, and can cause severe damage to the host tissues.

According to Dr Joanne Kurtzberg, director of the pediatric blood and marrow transplant program at Duke University Medical Center, the drug has saved a number of children’s lives and could increase the success rate of bone marrow transplants.

C. Randal Mills, CEO of Osiris, commented: “It’s really a good day for the concept and the hope behind stem cell therapies becoming a reality.”

The company intends to submit Prochymal for FDA approval in the same indication later this year, when it has gathered more data.

The use of adult stem cells avoids the ethical controversies surrounding the use of embryonic stem cells, offering a potential breakthrough for regenerative medicine.