What will likely become the world?s first approved stem cell cure for life-threatening disease is incubating in the Baltimore labs of Osiris Therapeutics Inc.
Osiris announced Thursday that it has received clearance from the Food and Drug Administration to begin an expanded treatment program for Prochymal. The approval will make the investigational stem cell product available to children with life-threatening graft vs. host disease, or GvHD, in which immune cells in transplanted marrow attack other cells.
“Prochymal has had a profound positive impact on the children that we have treated, all of whom had exhausted available therapeutic options,” said Dr. Paul Szabolcs of the pediatric Blood and Marrow Transplant Program at Duke University.
Osiris Chief Executive Officer C. Randal Mills told The Examiner he was confident the treatment would win FDA approval, which would make Prochymal the world?s first approved stem cell therapy.
The FDA streamlined the process for Osiris to provide Prochymal to children with GvHD. Prochymal, a formula based on adult immune system tissue called mesenchymal stem cells, is in Phase III clinical trials, the last stage of testing before FDA approval.
The company announced in December that early testing saved the lives of seven of 12 children with advance-stage GvHD, all of whom had been given fewer than 100 days to live.
“Since there are no approved treatments for GvHD and mortality is so high, gaining faster and more reliable access to Prochymal will be very helpful to the families we serve,” Szabolcs said.
The disease comes about when a tissue transplant, such as bone marrow, activates the body?s immune system against the recipient?s tissue.
Mesenchymal stem cells used in Prochymal are found at a ratio of one in every 100,000 bone marrow cells and are dispatched in the body to relieve inflammation and promote healing.
Osiris is seeking approval for treating three different conditions with Prochymal and has contracted with the Defense Department to investigate using the stem cells to fight radiation sickness.
