FDA Regulation of Stem Cell Therapies

The FDA's Center for Biologics Evaluation and Research regulates stem cell products as biologics under the Public Health Service Act, which means a product generally needs an approved Biologics License Application before it can be marketed for a specific disease claim. The agency's public list of approved cellular and gene therapy products runs to roughly 43 entries as of 2026, spanning CAR-T cancer therapies, gene therapies for genetic diseases, and a small number of stem cell products [1]. Within that list, the stem cell entries are almost entirely cord-blood-derived hematopoietic progenitor cell products: ALLOCORD, CLEVECORD, Ducord, and HEMACORD, along with several other cord blood bank products from institutions including the University of Colorado, MD Anderson, LifeSouth, and Bloodworks [1]. No mesenchymal stem cell, adipose-derived stem cell, or embryonic/iPSC-derived product currently holds a full FDA license.

A narrow regulatory exception, known as the 361 HCT/P pathway, lets certain minimally manipulated tissue products bypass full premarket approval if they meet strict criteria: minimal manipulation, homologous use, no combination with another article, and either no systemic effect or autologous use. Clinics offering same-day fat-derived stem cell injections have argued their products qualify under this exception or under a related 'same surgical procedure' exception. Courts have generally rejected these arguments when the processing goes beyond minimal manipulation or the use is not homologous to the tissue's normal function [2].

The clearest test of this question came in United States v. US Stem Cell Clinic, LLC. The FDA sued a Sunrise, Florida clinic after adipose-tissue-derived stem cell injections into the eyes of three patients with age-related macular degeneration left them blind [3][4]. The clinic removed fat tissue, isolated the stromal fraction containing stem cells, and injected it back into patients, arguing this fell under the 361 exception. A federal district court disagreed and issued a permanent injunction in June 2019, and the Eleventh Circuit Court of Appeals unanimously affirmed on February 11, 2021, ruling that the clinic's procedure did not qualify for either the 'same surgical procedure' or '361 HCT/P' exceptions and was therefore subject to full FDA drug regulation [3][5]. The ruling remains the leading appellate precedent establishing FDA jurisdiction over unproven stem cell clinic procedures nationwide.

FDA enforcement has continued steadily since that ruling, mostly through warning and untitled letters rather than litigation. In 2019 the agency sent an untitled letter to R3 Stem Cell, LLC of Scottsdale, Arizona and its CEO David Greene, flagging unapproved stem cell products marketed through more than 50 affiliated clinics nationwide for conditions including Lyme disease, diabetes, Parkinson's disease, stroke, kidney failure, and ALS [6][7]. More recently, the agency has focused heavily on exosome and umbilical-cord-derived products: a February 11, 2026 warning letter to Dynamic Stem Cell Therapy in Henderson, Nevada cited an umbilical-cord-derived product marketed for allogeneic use as an unapproved new drug and unlicensed biological product [8]. That letter was one of several issued between late 2024 and early 2026 to companies including Evolutionary Biologics, Chara Biologics, Supreme Rejuvenation, New Life Medical Services, and Innate Healthcare Institute, all following a similar pattern of flagging unapproved exosome or cord-derived biologics [9].

The FDA has also moved to make the legitimate approval pathway faster rather than only tightening enforcement on the illegitimate side. On September 24, 2025, the agency released draft guidance on expedited programs for regenerative medicine therapies for serious conditions, describing flexible clinical trial designs including adaptive trials, novel endpoints, and use of real-world evidence for products holding Regenerative Medicine Advanced Therapy designation [10][11]. RMAT designation itself, created by the 21st Century Cures Act, speeds up FDA interactions during development without constituting approval; recent RMAT grants include Orca Bio's Orca-Q for high-risk blood cancers and the iPSC therapy NouvNeu001 [12].

For consumers, the practical upshot is that the FDA's stance on stem cell clinics has hardened, not loosened, even as the agency works to speed up its own review pathway for products that actually complete rigorous trials. A clinic advertising an unapproved stem cell, exosome, or umbilical-cord product for a broad set of diseases is very likely operating outside FDA-sanctioned use, regardless of how the marketing describes the science. See our safety and red flags page for how to evaluate a specific clinic's claims.