The U.S. District Court for the District of Columbia previously held that an individual’s own cultured stem cells constituted both a drug under the Food, Drug, and Cosmetic Act (“FDCA”) and a biological product under the Public Health Service Act (“PHSA”) subject to FDA regulation.  U.S. v. Regenerative Sciences, LLC, 2012 WL 2989988 at *6 (D.D.C. July 23, 2012).  The Regenerative Sciences case is currently on appeal before the D.C. Circuit.  Opening briefs are due on December 10, 2012.

Should the court of appeals affirm the lower court’s decision, it will be a huge blow to personalized medicine.  Specifically, if one’s own stem cells are drugs when reinserted, and thus require new drug approval, it is difficult to conceive how any such products could pass clinical trials because each patient’s cells are particular to him or her.  As a result, FDA regulations will need to be amended in light of advances in personalized medicine using autologous stem cells.

At issue in the case is the Regenexx™ Procedure, which is for patients suffering from moderate to severe joint, muscle, tendon or bone pain.  The procedure starts with a licensed physician at a clinic using a needle to get a bone marrow sample from a patient’s hip.  Blood samples are also taken from the patient’s arm veins.  Both the marrow and the blood samples are sent to Regenerative Science’s lab, where the mesenchymal stem cells (“MSCs”) are separated from the bone marrow and grown to greater numbers using the natural growth factors in the patient’s blood.  The MSCs are then sent to a university genetics laboratory for quality assurance testing.  If the MSCs pass testing, they are put into syringes with an antibiotic and other additives.  The filled syringes are then sent back to the clinic where they are injected into the patient’s injured area, such as the patient’s knee, usually 4-6 weeks after they were removed.  The MSCs then repair the patient’s injured area over the course of several months.

According to the district court, the Regenexx™ Procedure met the statutory definition of a “drug” under the FDCA and a “biological product” under the PHSA because it is based on MSCs derived from a patient’s bone marrow and is intended for the treatment and mitigation of disease and injury.   Id.   It is easy to construe a biological product as a drug because both the definition of a “drug” and a “biological product” concern the prevention or treatment of disease.  See 42 U.S.C. § 262(i)(1) (defining a “biological product” as any “ . . . blood, blood component or derivative . . . or analogous product . . . applicable to the treatment or cure of a disease or condition in human beings”); 21 U.S.C. § 321(g)(1)(B) (defining “drug” as “articles intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease”).  Stem cell treatments should not be regulated as drugs, however.  Courts need to be mindful of the distinctions between drugs for the mass treatment of disease, for example, and procedures involving a single patient relying on the patient’s own stem cells.   Stem cell procedures are a practice of medicine which should not be subjected to drug pre-market approval requirements which are better suited for the manufacturing of drugs in mass.

Unfortunately, the district court declined to do so when it sided with FDA on the issue of whether the MSCs were minimally manipulated.  Specifically,  the court found that although the Regenexx™ Procedure was a human cell or tissue product (“HCT/P”), it was not a HCT/P that met the set of criteria listed in 21 C.F.R. § 1271.10.  HCT/Ps meeting section 1271.10’s criteria are only regulated under section 361 of the PHSA and Part 1271 of the CFR; HCT/Ps that do not meet the criteria, such as the Regenexx™ Procedure, are regulated as a drug, device, and/or biological product.”  21 C.F.R. § 1271.20.  One of the criteria of section 1271.20 is that the HCT/P be only “minimally manipulated.”  According to the court, the defendants’ admission that “[t]he processing of the cultured cell product involves many steps, including selective culture and expansion of a multitude of different types of blood-forming and rare bone marrow stromal cells using plastic flasks, additives and nutrients, and environmental conditions such as temperature and humidity, to determine the growth and biological characteristics of the resulting cell population,” supports FDA’s determination that the cells changed during the defendants’ process resulting in more than minimal manipulation of the patient’s HCT/Ps.   It is unclear how the characteristic of the cells changed, however, because the process is used to grow more MSCs, not change the nature of them using biotechnology for instance.

The district court also gave FDA broad authority to regulate the Regenexx™ Procedure under the Commerce Clause.  Despite the procedure taking place entirely intrastate, the court held that FDA had authority to regulate the Regenexx™ Procedure as a drug under the Commerce Clause because, among other things, the defendants combine an antibiotic that shipped through interstate commerce with the cell product before the drug is administered through patients with a syringe.  The D.C. Circuit must find that the Regenexx™ Procedure is not a drug for the district court’s Commerce Clause rationale not to apply.

In reaching its decision, the district court rejected the defendants’ argument that the Regenexx™ Procedure constituted the practice of medicine, and thus, was outside the scope of FDA’s jurisdiction.  The court reasoned that while the Food, Drug, and Cosmetic Act was not intended to regulate the practice of medicine, it was designed to control the availability of drugs that doctors use.  The court also distinguished between a doctor using an a FDA approved drug in an off-label way, which is allowed under the practice of medicine, and adding an approved antibiotic to a cell product to be injected into patients, which the court said causes the latter to be a drug with connections to interstate commerce.

As a result, FDA, for now at least, has the authority to regulate clinics offering stem cell treatments and require such clinics to follow good manufacturing practices.

If the D.C. Circuit does not reverse the lower court’s decision and stem cell procedures are regulated as drugs, stem cell pioneers will likely move to other countries with more favorable laws and there could be a subsequent uptick in medical tourism.

Share →

Leave a Reply