Asserting that “FDA may lawfully regulate the creation of a new drug product even if it contains some of a patient’s cells,” the Food and Drug Administration (FDA) has filed a final brief in an appeal pending before the D.C. Circuit Court of Appeals involving an enforcement action against a facility and several physicians who treat patients with a stem cell therapy that they claim is not subject to FDA regulation. United States v. Regenerative Sciences, LLC, No. 12-5254 (D.C. Cir., brief filed March 13, 2013).

The appeal was taken from a district court grant of FDA’s motion for summary judgment; the court agreed with FDA that the “defendants’ cultured cell product was a ‘drug’ because defendants intend their product to be used for the treatment of disease and injury” and that it was also “a ‘biological product’ under the Public Health Service Act.” The district court further agreed that the product did not qualify for regulation under rules applying to products that are minimally manipulated, and also determined that the “defendants had failed to comply with good manufacturing practice and failed to properly label their cultured cell product, thereby causing their product to be adulterated and misbranded.” The court permanently enjoined the defendants from future violations and ordered them to cease manufacturing their cultured cell product unless “they follow current good manufacturing practice in their laboratory and retain an expert to inspect their facilities.”

The drug at issue is developed from bone marrow or synovial fluid taken from a patient and sent to a laboratory along with the patient’s whole blood. The defendants then, according to FDA, “centrifuge the bone marrow or synovial fluid, and certain cells are removed. The removed cells are placed in a flask to incubate, along with the patient’s blood platelets, a nutrient solution, and other additives. The mesenchymal stem cells contained in this fluid adhere to the flask, and defendants remove them by applying Trypsin, an enzyme. Defendants harvest the cells and repeat the process, further culturing and expanding the cells over the span of two to three weeks.

“Defendants engage in this process in an attempt to isolate mesenchymal stem cells and ‘to determine the growth and biological characteristics of the resulting cell population.’ The resulting cell population is then combined with doxycycline, an antibiotic, ‘and other additives’ and placed in syringes.” The cultured cell product is then injected in the patients to treat “orthopedic conditions, such as non-healing bone fractures, osteoarthritis, injuries to the meniscus and rotator cuff, avascular necrosis (death of the bone tissue) of the shoulder and hip, and chronic bursitis.” FDA has not approved the drug. The agency’s expert stated that the processing “alters the original cells’ biological characteristics: For example, he explained that ‘[s]cientists have shown that bone-marrow derived cells that are cultured to manufacture [mesenchymal stem cells] change both in terms of their proteins and in the genes they express.’”

The appeal has not yet been scheduled for argument; the appeals court has also been asked to address findings that because component parts used in the processing are shipped in interstate commerce, the drugs can lawfully be regulated within Congress’s Commerce Clause authority, as well as challenges under the Administrative Procedure Act to an FDA rule adopted in 2001 and a statement of policy appearing in its preamble, which the defendants claim should have been subject to notice-and-comment rulemaking. FDA contends that it is too late to challenge the rulemaking.