World’s first Stem cell drug treatment approved

Osiris Therapeutics’s drug  Prochymal (remestemcel-L), for acute graft-versus host disease (GvHD), has been approved for marketing  by regulators in Canada.  The “drug” is approved for suppressing the immune system in patients that have received transplants, where the grafts attack the patients bodies.  This is a complication of bone marrow transplantation that kills up to 80% of children affected and currently steroids are used as first-line therapy with a success rate of only 30%-50%.

Prochymal, which demonstrated a clinically meaningful response at 28 days after therapy began in 61%-64% of patients, can now be used for use in children who have failed to respond to steroids. However, the approval is conditional on Osiris carrying out additional clinical trials to verify the anticipated benefit of the therapy.

Prochymal is an intravenous formulation of mesenchymal stem cells (MSCs), which are derived from the bone marrow of healthy donors between the ages of 18 and 30 years. The MSCs are grown in culture so that up to 10,000 doses of Prochymal can be produced from a single donor which are then stored frozen and infused without the need to type or immunosuppress the child.

More information can be found at the company website.

Portions  of this article were abstracted.

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