What the FDA Regulates About Stem Cells
In the United States, human cells, tissues, and cellular and tissue-based products — including most stem cell products — fall under FDA jurisdiction. Here's what that oversight actually covers.
The FDA's authority over stem cell products
Under the Public Health Service Act and the Federal Food, Drug, and Cosmetic Act, the FDA regulates human cells, tissues, and cellular and tissue-based products, commonly called HCT/Ps. This category includes bone marrow, cord blood, adipose (fat) tissue used for cellular therapy, and cell-based products derived from those sources.
When a stem cell product does not meet a narrow set of exemption criteria, the FDA classifies it as a biological drug. That means the product must have an approved Biologics License Application (BLA), or be administered under an active Investigational New Drug (IND) application, before it can be marketed or used in patients.
What FDA oversight covers
- Manufacturing standards — how the product is collected, processed, tested, and stored.
- Safety and effectiveness data — evidence from preclinical studies and controlled clinical trials.
- Labeling and marketing claims — what a manufacturer or clinic may say the product treats.
- Post-market monitoring — tracking adverse events after a product is approved.
- Facility inspections — for manufacturers and, in many cases, clinics administering the products.
What the FDA generally does not regulate
The FDA does not license individual physicians or the general practice of medicine — that is handled by state medical boards. However, the FDA does regulate the products those physicians use. A licensed doctor is still not permitted to administer an unapproved biological drug outside a clinical trial simply because they hold a medical license.
A narrow exemption exists
Some HCT/Ps are regulated only under Section 361 of the Public Health Service Act — a lighter-touch pathway that does not require pre-market approval. To qualify, a product must meet all four of the following criteria: it must be minimally manipulated, intended for homologous use, not combined with another article (with limited exceptions), and either have no systemic effect or, if it does, be for autologous use or use in a first- or second-degree blood relative. Products that fall outside these criteria are regulated as drugs. This distinction is covered in more detail in the 361 vs 351 guide.
Why this matters for patients
If a clinic offers a stem cell "treatment" for a condition like arthritis, multiple sclerosis, autism, or COPD, and the product is not approved by the FDA and is not being administered under an IND in a registered clinical trial, the offering likely falls outside FDA rules. That does not automatically mean the product is dangerous — but it does mean it has not been proven safe or effective for that use.