Title of Abstract
Stem cell based regenerative medicine therapies are being actively developed and hold great potential for treatment of a wide array of medical conditions. However, clinical use of stem cell-based products is novel and product characterization is challenging. In particular, identification of cell therapy product characteristics that predict in vivo performance of cell-based therapies remains challenging. In FDA terminology, lot release characteristics of identity, purity, and potency constitute critical quality attributes that are related to the ability of a given cell preparation to perform the desired biological function and result in the intended clinical effect. For stem-cell based regenerative medicine clinical trial proposals submitted to FDA, characterization of the cellular products often relies on the use of a small number of cell surface markers and some measures of cell product activity derived from current understanding of cells from the field of cell biology. The regulatory science question is whether or not the characteristics that are measured in product testing are indeed quality attributes and are predictive of clinical outcomes. The talk will describe FDA’s MSC Consortium and our research efforts to develop strategies that will result in cell characterization methods able to predict quality, potency, and safety of MSCs, with implications for other types of stem cell-based regenerative medicine products and other cell-based products in general.
Steven R. Bauer, Ph.D., is the chief of the Cellular and Tissue Therapy Branch (CTTB), Division of Cellular and Gene Therapies in the Office of Tissues and Advanced Therapies at the Center for Biologics Evaluation and Research (CBER) in the U.S. Food and Drug Administration. As the chief of CTTB, Dr. Bauer supervises CBER scientific staff engaged in review of cell-based biological therapies, policy development in emerging areas of cellular therapies, and research relevant to their use in clinical trials. His current research focuses on mesenchymal stem cell biology and stromal cell–hematopoietic cell interactions that influence the development of lymphocytes. Dr. Bauer received his Ph.D. in biochemistry from the University of Maryland in 1986. From 1986 through 1991, Dr. Bauer was a scientific member of the Basel Institute for Immunology in Basel, Switzerland. In 1991, Dr. Bauer joined CBER's Division of Cellular and Gene Therapies.