North America Legal and Regulatory Affairs
Mackenna Roberts, MAChoice TelemedLondon, United Kingdom Co-Editors:Crystal Ruff MBA, PhDTakeda PharmaceuticalsLondon, United Kingdom Joseph (Yossi) Schwartz MD, MPHMount Sinai Health SystemNew York NY, United States
Court ruling threatens FDA jurisdiction over autologous stem cell treatments
On August 30, 2022, a US federal judge in California issued a ruling against the FDA, finding that it does not have a legal basis to regulate and prevent clinics from providing experimental/unproven stem cell treatments.
US District Judge Jesus G. Bernal determined that multiple experimental stem cell treatments offered by California Stem Cell Treatment Center, Inc., Cell Surgical Network Corporation and its doctors do not fall within the legal definition of a ‘drug’ but instead qualify as surgical procedures or the practice of medicine, which is exempt from the FDA’s remit. It should be noted that Judge Bernal did find that one experimental cancer treatment was potentially subject to the Food, Drug and Cosmetics Act (FDCA) in his opinion, but the clinics and doctors voluntarily ceased offering it in 2017 (before the lawsuit commenced).
He concluded that the defendants “engaged in the practice of medicine, not the manufacture of pharmaceuticals”.
The decision directly conflicts with the precedent ruling in favour of the FDA in a similar case decided by a district judge in Florida, which was affirmed on appeal by the federal Eleventh Circuit Court in June 2021. Judge Bernal acknowledged but departed from the 2021 Court of Appeal precedent for reasons set out in his 19-page ruling.
This leaves courts, regulators and legitimate cell therapy innovators in a state of confusion. Judicial opinion is divided and future courts will have to decide which decision to follow. This case will likely be appealed but until then the question of whether the FDA has the legal right to regulate these treatments, as it claims, is unclear with two opposite interpretations of the FDCA regulations.
What is the problem?
As Judge Bernal rightly described, the line between a ‘drug’, which is regulated by the FDA, and a ‘medical procedure’, which is not, is “especially muddy” in relation to stem cell procedures involving stromal vascular fraction cells (SVF) and expanded mesenchymal stem cells (MSCs) derived from adipose tissue involving the extraction, isolation and reinfusion of the patient’s own cells.
At the center of the debate between the FDA and clinics is the question of whether the SVF cells and MSCs are exempt from being regulated as a type of biologic drug, specifically as a human cells, tissues, or cellular or tissue-based products (HCT/Ps). The critical threshold is the degree to which the cell is manipulated (ie important characteristics changed) in the extraction process. Clinics argue that no new product is created that is not already naturally occurring in the person, and the FDA argues these are more than minimally manipulated because the extraction process eliminates the components in the tissue that allow it to serve its structural function as fat.[i]
The decision comes at a time when clinics have proliferated offering direct-to-consumer costly, experimental and unproven stem cell treatments for a range of conditions from arthritis to strokes, cancer and autism. As they are unproven, these could be ineffective or even dangerous. Three patients were blinded by an experimental stem cell treatment for acute macular degeneration in 2017 by way of example.
This decision hinders the US government’s ability to regulate these clinics and protect consumers from unproven, expensive stem cell treatments. ISCT has responded to the federal ruling with a written statement that expresses its concern about the decision and highlights scientific inaccuracies in the judgment that may have led to this decision.
For more information see:
FDA - Guidance Documents
Cellular & gene Therapy Guidances
United States v. California Stem Cell Treatment Center Inc. et al., 5:18-cv-01005, in the US District Court for the Central District of California
United States v. US Stem Cell Clinic, 403 F.Supp. 3d 1279, 1296-98 (S.D. Fla. 2019)
United States v. US Stem Cell Clinic, LLC, No. 19-13276 (11th Cir. 2021)
[i] Regulatory Considerations for Human Cells, Tissues, and Cellular and Tissue-Based Products: Minimal Manipulation and Homologous Use; Guidance for Industry and Food and Drug Administration Staff
This newsletter includes links to issues and activities that might be of interest both on the Health Canada as well as the FDA websites. Please note that you can follow HC and FDA on Facebook and Twitter. You can also subscribe to receive emails from the FDA at https://www.fda.gov/about-fda/contact-fda/get-email-updates
What’s new in Biologics, radiopharmaceuticals and genetic therapies can be found at: https://www.canada.ca/en/health-canada/services/drugs-health-products/biologics-radio-pharmaceuticals-genetic-therapies/what-new-biologics-radiopharmaceuticals-genetic-therapies-health-canada.html
Information for health product manufacturers and distributors in relation to COVID-19: https://www.canada.ca/en/health-canada/services/drugs-health-products/covid19-industry.html
What’s new for Biologics including Approval and Determination Letters:
COVID-19 Information and Resources:
Updated Approvals and Listings:
Complete List of Licensed Products and Establishments
Complete List of Substantially Equivalent 510(k) Device Applications
Complete List of Currently Approved Premarket Approvals (PMAs)
Complete List of Currently Approved NDA and ANDA Application Submissions
2021 Biological Approvals