FDA Stem Cell FAQ

Ten common questions about how the FDA regulates stem cell therapies in the United States, answered in plain language.

Are stem cell therapies FDA-approved?

Only a limited set of stem cell products is FDA-approved, primarily hematopoietic stem cell transplants for certain cancers and blood disorders, licensed cord blood products for hematopoietic reconstitution, and some cell-based gene therapies (such as certain CAR T-cell products) for specific cancers. Most stem cell treatments advertised for conditions like arthritis, autism, or anti-aging are not FDA-approved.

Does the FDA regulate stem cell treatments at all?

Yes. The FDA regulates human cells, tissues, and cellular and tissue-based products (HCT/Ps), including most stem cell products, under the Public Health Service Act and the Federal Food, Drug, and Cosmetic Act. Clinics that claim the FDA does not regulate their stem cell products are typically incorrect.

What is the difference between a 361 and a 351 product?

A 361 HCT/P is regulated only for communicable-disease prevention and does not need pre-market approval, but it must meet strict criteria (minimally manipulated, homologous use, not combined with other articles, and either non-systemic or autologous/close-relative use). Any HCT/P that fails these criteria is regulated as a 351 biological drug and requires an approved BLA or an active IND.

Are stem cell injections for knee arthritis FDA-approved?

No. As of this writing, the FDA has not approved any stem cell product for knee osteoarthritis. Clinics offering such injections are administering an unapproved product for that use, or the product is being studied under an IND in a registered clinical trial.

Are umbilical cord, amniotic, or exosome products FDA-approved?

The FDA has approved certain umbilical cord blood products for hematopoietic reconstitution, but not for the wide range of conditions marketed by consumer clinics. Amniotic fluid and exosome products marketed for injection into joints, blood, or the nervous system are generally considered unapproved 351 products by the FDA, and the agency has issued warning letters accordingly.

Is it legal for a licensed doctor to give me an unapproved stem cell product?

State medical boards regulate the practice of medicine, but the FDA regulates the products physicians use. A licensed doctor may not lawfully market or administer an unapproved biological drug outside an authorized IND simply because they hold a medical license.

How do I know if a stem cell trial is legitimate?

Legitimate trials have an NCT number on ClinicalTrials.gov, a named sponsor, IRB approval, a formal protocol, and an informed consent process. In most legitimate trials, participants are not charged for the investigational product itself. If a clinic cannot provide these details, treat it as a red flag.

What are the risks of unapproved stem cell products?

The FDA has documented infections (including bloodstream and joint infections), permanent vision loss following intraocular injections, tumor formation, pulmonary emboli, and severe allergic reactions among patients who received unapproved stem cell products.

How do I report a problem with a stem cell treatment?

Patients and providers can report adverse events through the FDA's MedWatch program at fda.gov. Reporting helps the FDA identify patterns and take action against clinics or manufacturers causing harm.

Where can I find the official FDA list of approved cellular therapy products?

The FDA maintains a public list at fda.gov under 'Approved Cellular and Gene Therapy Products'. That list is the authoritative source for what is actually approved in the United States.