D.C. Circuit Upholds FDA Regulation of Autologous Stem Cell Therapies

The FDA continues to monitor the emerging field of stem cell medicine, which offers the possibility of treating certain medical disorders by the application of new cells which replenish deteriorating tissues (e.g., Parkinson’s, heart disease, diabetes). The prospect of harvesting a patient’s own stem cells and administering them to sites of cellular stress is a new paradigm for disease treatment (autologous stem cell therapy). Nonetheless, such practices challenge existing legal regimes which regulate the introduction of pharmaceutical and biologic products into the marketplace. Regenerative Sciences, Inc., is a Colorado corporation that its Regenexx stem cell treatment, offered to patients suffering from a number of musculoskeletal conditions. The procedure involves withdrawing bone marrow from a patient, extracting the mesenchymal stem cells from the marrow, and processing the cells to create a therapeutic preparation for injection to a site of interest, with a goal of restoring function to impaired joints. Since 2008, the FDA has cautioned the company that its practice was likely violating the Federal Food Drug and Cosmetic Act (FDCA) because it involved the use of an unapproved drug and likely violating the a biological product under the Public Health Service Act (PHSA), because it used an unapproved biologic product. The FDA also inspected the Regenerative facilities in 2009, and found numerous departures from good manufacturing practices. The FDA then sued to enjoin the company from offering unapproved stem cell treatments and prevailed in federal district court in 2012. The court granted summary judgment on a motion by the FDA that the Regenerative stem cell preparation was both a drug and a biologic, and, as such, was both adulterated and misbranded, in violation of both statutes (see earlier post). The court issued a permanent injunction against Regenerative, prohibiting it from offering the stem cell treatment. 

Now, the D.C. Circuit has upheld the district court’s decision, rejecting the defendant’s arguments that it was simply engaged in the practice of medicine, which is not regulated by the FDA, and is governed by state law. The appellate court affirmed the lower court’s finding that the stem cell preparation was both a “drug” and a “biologic” and required FDA approval before use in clinical medicine, noting that these existing laws do not interfere with the practice of medicine:

Appellants’ construction of the FDCA, by contrast, would allow states to gut the FDCA’s regulation of doctors, and thereby create an enormous gap in the FDCA’s coverage, by classifying the distribution of drugs by doctors as the practice of medicine. Given Congress’s intent that the FDCA’s “coverage be as broad as its literal language indicates," United States v. An Article of Drug . . .Bacto-Unidisk, 394 U.S. 784, 798 (1969), such a construction is not tenable. 

Critics of these FDA enforcement activities argue that the application of the standard drug approval process to stem cell therapies (especially with the use of autologous cells harvested from the patient) undercuts innovation in the field. Proponents of FDA regulation argue that existing statutory responsibilities call for the agency to monitor the use of stem cell therapies, yet some concede that the use of autologous stem cell therapies calls for a novel regulatory policy that is especially tailored to these procedures, rather than the application of “one size fits all” FDCA and PHSA requirements.