MSCs: The US regulatory perspective

Abstract

In the USA, the Food and Drug Administration (FDA - the Agency) regulates cellular therapies, primarily through the Center for Biologics Evaluation and Research (CBER) Office of Cellular, Tissue and Gene Therapies. The rapid expansion of these therapies has prompted the Agency both to determine the applicability of existing regulations and to develop specific new laws. The strategy that has evolved is based upon perceived risks to the donor and recipient of the cell product and to the product itself by ex vivo manipulation during the manufacturing process. Mesenchymal stromal cell (MSC) products are considered to be more-than-minimally manipulated, due to the requirement for expansion of the cells in culture. As such, the product must be manufactured under current Good Manufacturing Practices (cGMP) and clinical trials carried out under an Investigational New Drug (IND) application. The development of this regulatory strategy and the factors involved in cGMP manufacturing and applying for an IND are reviewed in this chapter.