FDA regulation of stem-cell-based therapies.

In the interest of public safety, the Food and Drug Administration (FDA) has jurisdiction over the production and marketing of any stem-cell–based therapy involving the transplantation of human cells into patients. The FDA’s recently promulgated regulations regarding human cells, tissues, and cellular and tissue-based products1 provide an appropriate regulatory structure for the wide range of stem-cell–based products that may be developed to replace or repair damaged tissue. Basic and clinical scientists, as well as scientists working in the biotechnology and pharmaceutical industries, need an increased awareness of the questions that must be answered before a stem-cell–based product can be used clinically. Unlike pharmaceutical products, many stem-cell– based products may originate in academic laboratories where researchers are unfamiliar with the applicable regulations. We outline here the existing regulations regarding cell and tissue products that the FDA is likely to apply to the preclinical development and testing of various types of stem-cell–based therapies. We also make specific recommendations about how scientists should address the inherent safety and efficacy issues associated with these therapies.