Prochymal®, mesenchymal stem cells for treatment of graft versus host disease

Prochymal®, an intravenously administered formulation of mesenchymal stem cells (MSCs), is being evaluated in Phase 3 clinical trials for acute graft versus host disease (GvHD). Prochymal can be infused safely into adult patients with acute GVHD and induces responses in a high proportion of GvHD patients. Prochymal® was also evaluated in children. No infusional or other identifiable acute toxicity was seen in any patient. Multiple infusions of MSCs were well tolerated and appeared to be safe in children. Clinical responses, particularly in the gastrointestinal system, were seen in the majority of children with severe refractory acute GvHD. Given the favorable results observed in a patient population with an otherwise grave prognosis, it was concluded that MSCs bear potential for the treatment of acute GvHD. Overall 61% of pediatric patients responded to treatment with Prochymal® at day 28 and 77% at 100 days post infusion of MSCs. Additional treatment induced more response and complete resolution in some.