Stem cells facilitate graft-versus-host response
On Thursday, May 17, Osiris Therapeutics announced that it had received official permission from the Ministry of Health of Canada (Health Canada) for the commercial use of the drug Prochymal (remestemcel-L), intended for the treatment of "graft versus host reaction" (GVHD) in children. The uniqueness of this drug lies in the fact that its active ingredient is adult stem cells. This historic decision is the first official approval of a finished stem cell drug in Canada.
"Graft versus host reaction" is the main complication of bone marrow transplantation. The associated mortality of pediatric patients is up to 80%, while the death of patients often occurs within a few weeks after the first symptoms appear.
Prohimal is a preparation of mesenchymal stem cells isolated from the bone marrow of healthy donors aged 18-30 years. These cells are cultured in the laboratory using a technique that allows to obtain up to 10,000 doses of the drug from the material of one donor.
The finished drug is intended for storage in frozen form in medical institutions and subsequent intravenous administration to stop the acute "graft versus host reaction" without the need for preliminary immunological typing and selection of donor cells.
The decision on the approval of prohimal was made based on the recommendations of an independent commission of experts authorized to evaluate the safety and effectiveness of the drug. During the phase III clinical trial involving children with severe refractory (not responding to standard therapy) graft versus host reaction, 61-64% of patients showed clinically significant improvements within 28 days after the start of treatment. Moreover, prochymal therapy provided a significant increase in the survival rate compared to the historical control group, which included pediatric patients with similar posttransplantation reactions. According to this indicator, the effectiveness of the drug was most pronounced in the treatment of patients with the most severe forms of the graft-versus-host reaction.
As a condition for obtaining official approval, the clinical benefits of using prohimal in the future have yet to be evaluated as part of a control study of comparable cases, and all patients receiving the drug will be invited to join a special register to monitor the long-term effects of therapy.
To date, for the treatment of a graft-versus-host reaction that does not respond to steroid drugs in children, prohimal is available in several countries, including the USA. At the end of this year, it should appear on the Canadian market.
Currently, the drug is also undergoing phase III clinical trials as a treatment for refractory Crohn's disease and is at various stages of clinical testing as a drug for the treatment of myocardial infarction and type 1 diabetes.
Evgeniya Ryabtseva
Portal "Eternal youth" http://vechnayamolodost.ru based on the materials of the Osiris Therapeutics press release:
World's First Approved Stem Cell Drug; Osiris Receives Marketing Clearance from Health Canada for Prochymal.
21.05.2012