There may be hope for some desperate patients that don’t respond well to first line steroid treatment, as the first stem cell drug lands a conditional approval in Canada.
The company that gained approval is Osiris Therapeutics Inc, which is based in Columbia, Maryland. They have just announced that Canadian health regulators have approved its treatment for acute graft-versus host disease in children, making it the first stem cell drug to be approved for a systemic disease anywhere in the world.
Graft versus host disease (GvHD) is a potentially deadly complication from a bone marrow transplant, when newly implanted cells attack the patient’s body. Symptoms range from abdominal pain and skin rash to hair loss, hepatitis, lung and digestive tract disorders, jaundice and vomiting. This happens most often when the donor is not an exact match for the recipient.
The disease kills up to 80 percent of children affected, Osiris said. To date there have been no approved treatments for the disease. Canadian authorities approved the therapy, Prochymal, for use in children who have failed to respond to steroids.
“It’s really a good day for the concept and the hope behind stem cell therapies becoming a reality,” C. Randal Mills, the chief executive of Osiris, said in an interview.
Prochymal is made up of bone marrow stem cells derived from an adult donor and is designed to control inflammation, promote tissue regeneration and prevent scar formation. The stem cells are separated out from the marrow and expanded in culture, so that one donation is enough to make as many as 10,000 doses.