《MSC-Based Product Characterization for Clinical Trials An FDA Perspective》.pdfVIP

Cell Stem Cell Forum MSC-Based Product Characterization for Clinical Trials: An FDA Perspective Michael Mendicino,1,2,* Alexander M. Bailey,3 Keith Wonnacott,2 Raj K. Puri,2 and Steven R. Bauer2,* 1Office of the Commissioner (OC), Office of the Chief Scientist (OCS), Office of Regulatory Science and Innovation (ORSI), 10903 New Hampshire Boulevard, Silver Spring, MD 20993, USA 2Center for Biologics Evaluation and Research (CBER), Office of Cellular, Tissue and Gene Therapy (OCTGT), Division of Cell and Gene Therapy (DCGT), 1401 Rockville Pike, Rockville, MD 20852, USA 3CBER, OCTGT, Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT), 1401 Rockville Pike, Rockville, MD 20852, USA *Correspondence: m.mendicino.phd@ (M.M.), steven.bauer@ (S.R.B.) /10.1016/j.stem.2014.01.013 Proposals submitted to the FDA for MSC-based products are undergoing a rapid expansion that is charac- terized by increased variability in donor and tissue sources, manufacturing processes, proposed functional mechanisms, and characterization methods. Here we discuss the diversity in MSC-based clinical trial prod- uct proposals and highlight potential challenges for clinical translation. Introduction reviews each regulatory submission product IND submissions to the FDA MSCs, commonly referred to as mesen- based on its own merits, and nomencla- between 2006 and 2012 in the set we chymal stem cells or mesenchymal stro- ture is not a regulatory concern during assessed. In this period, there was also mal cells, are a diverse population of cells early clinical development. Closer to licen- a substantial increase in registered MSC with a wide range of potential therapeutic sure, however, nomenclature assumes clinical trials initiated worldwide (246 tri- applications. Despite considerable inter- greater significance for regulatory