Stem cell-based cellular therapies are being actively developed and hold tremendous promise for treating a wide variety of medical conditions, from diabetes to heart disease and joint repair.  However, the use of stem cell-based products is new and characterizing the product is challenging.  Specifically, FDA is conducting research into identifying cell therapy product characteristics that will predict the reliably of the performance of cell-based therapies in humans.  In many cases, this is a challenge that is largely unresolved. 

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. 

For many investigational stem-cell based products, we currently 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 is whether or not the characteristics that are measured in product testing are predictive of clinical outcomes.  This presentation will describe FDA’s multipotent stromal cell (MSC) Consortium and our research efforts to develop strategies that will result in cell characterization methods that can predict quality, potency, and safety of MSCs.  This could have implications for other types of stem cells and cell-based products in general.