Stem Cell Therapy: Separating Reality From Hype

The science

What Stem Cells Actually Are

Stem cells can develop into specialized cell types and, in principle, support tissue repair — which is the basis of genuine scientific excitement about the field. That promise is real and the subject of serious ongoing research.

But promise is not the same as proven, approved treatment, and the gap between the science's potential and what commercial clinics sell is wide.

Featured

Featured doctors

Featured doctors: Dr. Vivek Mohan, a board-certified, fellowship-trained orthopaedic spine surgeon at the Orthopaedic Spine Institute in Hinsdale, Illinois, who specializes in minimally invasive and endoscopic spine surgery; Dr. Alexandra Conde Green, a double-trained plastic surgeon at ACG Plastic Surgery in Boca Raton, Florida, known for her work with regenerative cells in procedures such as fat grafting; and Dr. Alexander Golberg, a New York physician whose Park Avenue practice, Dr. Golberg Functional & Aesthetic Medicine, focuses on regenerative, anti-aging and aesthetic medicine and chronic pain management. See all regenerative medicine physicians, orthopedics guides or find a doctor.

Haute MD members are featured through paid membership. Membership is not an endorsement or a ranking.

What's approved

What's Actually FDA-Approved

FDA keeps a public list of approved cellular and gene therapy products (last updated September 17, 2026). The approved products that contain stem, progenitor or stromal cells fall into four groups:

• ·Cord blood (hematopoietic progenitor cells). Nine licensed cord blood products are used in unrelated-donor transplants for disorders of the blood-forming system. A modified cord blood product, Omisirge, is approved for certain blood cancers and severe aplastic anemia.
• ·Gene therapies made from your own blood-forming stem cells. Casgevy and Lyfgenia (sickle cell disease), Zynteglo and Casgevy (transfusion-dependent β-thalassemia), Skysona (cerebral adrenoleukodystrophy), Lenmeldy (metachromatic leukodystrophy), Waskyra (Wiskott-Aldrich syndrome) and Kresladi (leukocyte adhesion deficiency-I). Each is approved for a defined patient group, based on clinical-trial data.
• ·Transplant-support therapy. Tregzi, approved in 2026, combines donor regulatory T cells with hematopoietic stem and progenitor cells for matched-donor transplants in adults with blood cancers.
• ·Mesenchymal stromal cells. Ryoncil, approved in December 2024, is made from donor bone marrow and is approved for steroid-refractory acute graft-versus-host disease in children 2 months and older.

None of these is approved for orthopedic, anti-aging, hair or cosmetic use. Don't confuse stem cells with MACI, an FDA-approved implant grown from a patient's own knee cartilage cells (chondrocytes, not stem cells) for knee cartilage defects; see What Is MACI Knee Cartilage Repair?.

The rules

"FDA-registered" vs. FDA-approved: the Section 361 rule

Many clinics say their product is "FDA-registered" or "361-compliant." Here is what that means. Federal rules (21 CFR Part 1271) let a narrow group of human cell and tissue products (HCT/Ps) skip premarket approval and be regulated "solely under section 361" of the Public Health Service Act, but only if the product meets all four criteria:

• ·It is minimally manipulated: processing doesn't alter the cells' relevant biological characteristics.
• ·It is for homologous use: it performs the same basic function in the recipient as in the donor.
• ·It isn't combined with another article, apart from water, crystalloids or a sterilizing, preserving or storage agent.
• ·It has no systemic effect and doesn't depend on living cells' metabolic activity for its main function, or, if it does, it's used in the same person (autologous), in a first- or second-degree blood relative, or for reproduction.

Those firms must register with FDA and list their products, but registration is not approval. A separate exception covers removing tissue and putting it back into the same patient during the same surgical procedure. A product that fails any of the four tests is regulated as a drug or biologic. It needs an approved biologics license to be sold, and an active investigational new drug application (IND) to be used in a clinical trial. FDA ended its grace period for these products on May 31, 2021. Since then, it has said a product that needs approval but lacks it "may not be lawfully marketed," even while an IND is pending.

What about "RMAT"? Regenerative Medicine Advanced Therapy designation, created by the 21st Century Cures Act, is an expedited-review status FDA can grant an investigational cell therapy for a serious or life-threatening condition when early clinical evidence suggests it could meet an unmet need. It is requested under an IND, and it is not an approval. Products regulated solely under Section 361 aren't eligible.

What's marketed

What Clinics Are Actually Selling

In March 2021, a study in Cell Stem Cell found 1,480 U.S. businesses operating 2,754 clinics selling purported stem cell treatments that aren't FDA-approved, more than four times as many businesses as five years earlier. A 2020 study cited in PNAS (2026) found that 96% of clinic websites contained at least one misstatement. FDA has specifically warned about unapproved products sold as stem cells, stromal vascular fraction (fat-derived cells), umbilical cord blood, Wharton's jelly, amniotic fluid, "ortho-biologics" and exosomes. It continues to send warning letters, including one in February 2026 to a Nevada clinic marketing an umbilical cord-derived product for knee, shoulder and hip injuries.

The risks

The Documented Risks

FDA lists these safety concerns with illegally marketed regenerative products: blindness; tumor formation; neurological events; bacterial infections, including life-threatening blood infections; reactions where cells are collected or injected; unwanted inflammatory or immune responses; cells moving elsewhere in the body and growing into the wrong tissue type; contamination during processing or untested donor material; and failure of the therapy to work when an approved treatment was available.

How to decide

How to protect yourself

Legitimate access to an investigational stem cell therapy is generally through a clinical trial run under an FDA IND. FDA cautions that a ClinicalTrials.gov listing or an "FDA-registered" label does not by itself mean a product is legally marketed, and that if you're being charged for an unapproved product, or offered one outside a clinical trial, "you are likely being deceived." Before any treatment, ask for the FDA-issued IND number and the official FDA acknowledgment. Report a bad reaction to FDA MedWatch, and send questions to ocod@fda.hhs.gov. Don't let hope for an unproven treatment delay care that works today.

Florida

Florida's stem cell law

Since July 1, 2025, Florida law (sections 458.3245 and 459.0127 of the Florida Statutes) has allowed licensed physicians to perform stem cell therapy that is not FDA-approved, if it's within their scope of practice and related to orthopedics, wound care or pain management. The cells must come from an FDA-registered facility accredited by one of the listed organizations, with a post-thaw viability report for the lot. Advertising must carry a notice that the physician performs stem cell therapies "that have not yet been approved by the United States Food and Drug Administration," and patients must sign a consent form that says so and describes the risks and alternatives. The law doesn't make any product FDA-approved. If you're considering treatment in Florida, ask to see the notice, the consent form and the facility's accreditation.