This study reports the two-year safety and efficacy outcomes of a clinical trial involving 15 participants with steroid-resistant acute graft-versus-host disease (GvHD) treated with iPS cell-derived mesenchymal stromal cells (CYP-001). At the two-year mark, 60% of participants survived. Deaths were attributed to common complications in allogeneic hematopoietic stem cell transplantation recipients and were not considered treatment-related. No serious adverse events, tumors, or other safety concerns associated with CYP-001 were observed. The results suggest that systemic delivery of iPS cell-derived cells is safe and well-tolerated over a two-year period.

Kilian Kelly, Adrian J. C. Bloor, James E. Griffin, Rohini Radia, David T. Yeung, John E. J. Rasko