The drug has been approved for graft versus host disease (GVHD), which can develop after a bone marrow or stem cell treatment. Instead of the the patient's body rejecting the foreign material, the foreign material attacks the healthy cells within the host, causing a slew of terrible symptoms. The disease presents when there isn't a perfect match between donor and recipient, and incidence can rise as high as 80% in unrelated donors.
Prochymal is made up of mesenchymal stem cells from the bone marrow of healthy donors. The cells are grown and expanded to create thousands of doses from each donor. The treatment is only approved in children, and only then for severe cases. The company needs more data before submitting Prochymal to the FDA for US approval, which could happen later this year.
The commercial success of Prochymal will likely be closely watched, as it is one of the earliest allogeneic stem cell treatments that has been approved for commercial use. Determining whether the therapy will be reimbursed in the US could also set a precedent for other soon-to-be-approved treatments.
Learn about other stem cells treatments working their way through phase 3 trials at the Stem Cells USA and Regenerative Medicine Congress 2012!