Regulatory hurdles for stem cells

One of the big surprises to me from the stem cell meeting in San Francisco over the weekend was the academic's astonishment at how much work is involved in getting a treatment approved by the Food and Drug Administration (FDA). If stem cell treatments are to be patented and sold like prescription drugs which is a likely commercial goal then a very long process is involved and the steps are many. The steps are also highly regulated by authorities, including the U.S. FDA, the European Agency for the Evaluation of Medicinal Products (EMEA), and the Korean Food and Drug Administration (KFDA).

Human clinical studies can begin in another country or in a U.S. state as long as no international or interstate commerce is involved an unlikely situation given the limited sources of stem cells. To conduct studies that may eventually be used to support a marketing application, an Investigational New Drug Application (IND) is necessary and this requires extensive, highly regimented animal (preclinical) studies. Essentially, the group planning to introduce the stem cells into humans has to 1) characterize the material to be used, 2) demonstrate that the material is unlikely to cause safety problems, and 3) provide a plan for Phase I clinical studies.

A natural question to be asked here is, why hasn't this been necessary with bone marrow transplants? The issue is profit. A group, such as a particular hospital, can perform medical procedures considered beneficial for patients without all the FDA regulatory process as long as the procedure is not patented and sold for profit. That the facility performing the procedure is likely to make a profit because patients will go there rather than elsewhere is a different issue. Thus, we have bypass surgery for heart conditions as an accepted medical procedure, but balloons inserted into blood vessels to increase blood flow were patented and met FDA regulations as medical devices.

So, as we consider the future of stem cell research we also have to include the American free-market system and the need for companies to make a profit. What I don't know and perhaps a reader can tell us what regulations companies that offer fetal stem cell treatments or cord blood cell treatments have to follow.

Marie Godfrey, PhD

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