Stem cells, like other therapies intended to treat, cure, or prevent disease, require U.S. Food and Drug Administration (FDA) approval before they can be marketed. So far, the FDA has only approved the use of cord blood-derived hematopoietic progenitor cells. Hemacord (HPC, cord blood) is manufactured by the New York Blood Center for patients with disorders affecting the body’s blood-forming system.
The FDA’s Center for Biologics Evaluation and Research regulates human cells, tissues, and cellular and tissue-based products (HCT/P) that are intended for implant, transplant, infusion, or transfer into a human recipient, including hematopoietic stem cells.
Requirements aim primarily to reduce chances for the spread of disease. To distribute medical products that derive from stem cells, the FDA requires an intense review process in which manufactures must show how the product is made, and that it is safe and effective.
A new FDA draft guidance, titled “Homologous use of Human Cells, Tissues, and Cellular and Tissue-based products,” includes language concerning stem cells. Find it at www.regulations.gov.
The recommendations address healthcare providers; businesses that produce human cells, tissues and cellular and tissue-based products (HCT/Ps); and FDA staff.
Under the proposed guideline, homologous use refers to medical uses for HCT/Ps, including stem cells, that generally mirror how the product was used in the donor and how it will be used in the recipient — even if the recipient is also the donor.
For example, according to the proposed guideline, cells or tissues that come from skin must be used to repair, reconstruct, replace, or supplement the recipient’s skin. But pancreatic islet cells transplanted to the liver for endocrine function after pancreatectomy is also considered homologous use because regulation of glucose is a function of pancreatic islets.
The guideline does not allow a HCT/P to be used for myriad diseases or conditions based on the presumption that the product would not have been produced with a homologous (specific) use intended.
The FDA remains concerned for people with disabling conditions and has high hopes for cures that are not yet available. The agency warns against providers of stem cell treatments that may be illegal and potentially harmful, and they urge people who travel out of the U.S. for stem cell treatments to be well-advised concerning treatment regulations in other countries.
According to an FDA consumer information release: “While stem cell therapies theoretically have potential promise, their use also raises a number of safety concerns including: the ability of the cells to migrate from the site of administration and differentiate (or change) into inappropriate cell types; excessive cell growth; and the development of tumors. These safety concerns must be addressed as part of the risk/benefit evaluation of these products.”
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