73 - Stem Cell-Based Products in Medicine: FDA Regulatory Considerations

Abstract

This chapter describes development of Food and Drug Administration (FDA) recommendations and summarizes current recommendations applicable to stem cell-based cellular therapies. FDA recommendations for stem cell based product manufacture and characterization incorporate the tremendous experience gained in many cell therapy clinical trials and the experience of the entire field of stem cell research. The challenges of translating the promise of stem cells from successful basic scientific research into clinical use are great, but the FDA is prepared to work with the scientific community to bring safe and effective stem-cell therapies into the medical armamentarium. The FDA regulatory approach requires multidisciplinary expertise to evaluate proposed clinical trials and includes reviewers with knowledge of medicine, pharmacology/ toxicology, cellular therapies, and cell biology. Accordingly, the Office of Cellular, Tissue, and Gene Therapies (OCTGT) employs scientists and clinicians involved in full-time review and some who, in addition to their review responsibilities, also pursue laboratory research programs in areas, such as developmental biology. A major goal of FDA oversight of cellular therapies is to ensure safety of patients who receive an investigational product. Because direct measurement of stem cell potency may be challenging due to lack of appropriate in vitro or in vivo assays, use of other means to demonstrate potency could be adequate for initiation of clinical trials.