Mesenchymal Stromal Cells and the Approach to Clinical Trial Design: Lessons Learned From Graft Versus Host Disease

Mesenchymal stromal cells have shown enormous promise as a prototypic cellular therapy for a number of diverse indications including regeneration of damaged connective tissue, cardiovascular diseases, pulmonary diseases, neurologic disorders, and immune-mediated disorders. Despite the broad interest in this promising therapy, translational development of mesenchymal stromal cells is proving to be difficult. Focusing on graft versus host disease, which is the indication closest to clinical approval, this review will discuss some of the challenges facing clinical approval and development. Ultimately, deeper understanding of the underlying mechanism of action will allow us to choose the most potent and effective product or by-product for the next generation of studies.