Thursday, Mar. 8, 2018
12:00 p.m. ‒ 1:00 p.m. EST
Register here for the webcast: CE Credit available.
Steven Bauer, Ph.D.
Chief, Cellular and Tissue Therapy
Division of Cellular and Gene Therapies
FDA’s Center for Biologics Evaluation and Research
Stem cell-based cellular therapies hold tremendous promise for treating a wide variety of medical conditions, from repairing heart, nerve, and brain damage to treating diabetes. However, the use of stem cell-based products is new and characterizing the product still faces hurdles. FDA’s Dr. Steven Bauer and his lab are conducting research into identifying cell therapy product characteristics that will predict the reliably of the performance of cell-based therapies in humans.
FDA is concerned with lot release characteristics of identity, purity, and potency, which constitute quality attributes. Ideally, these attributes are related to a given cell preparation’s ability to perform the desired biological function--and result in the intended clinical effect.
But for many investigational stem-cell based products, we don’t know if the measurements we use to characterize products will predict their clinical effectiveness. For stem-cell-based clinical trial proposals submitted to FDA, the products are characterized using a small number of cell surface markers and simple measures of cell product activity.
The regulatory science question before FDA is whether or not the characteristics that are measured in product testing are predictive of clinical outcomes. Dr. Bauer’s presentation will describe FDA’s multipotent stromal cell (MSC) Consortium and his research efforts to develop strategies that will result in cell characterization methods that can predict quality, potency, and safety of MSCs. This research could have implications for other types of stem cells and cell-based products in general.