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FDA Stem Cell Guide

Currently FDA-Approved Uses of Stem Cells

The list of FDA-approved stem cell products is much shorter than online marketing suggests. Here is the general shape of what is actually approved.

Hematopoietic stem cell transplantation

The most established approved use of stem cells is hematopoietic stem cell transplantation (HSCT), sometimes called a bone marrow or cord blood transplant. HSCT uses blood-forming stem cells — typically collected from bone marrow, peripheral blood, or umbilical cord blood — to rebuild a patient's blood and immune system.

It is used to treat certain cancers and disorders of the blood and immune system, such as leukemias, lymphomas, some inherited immune deficiencies, and certain genetic disorders of blood cell production. HSCT is a rigorous medical procedure typically performed at specialized transplant centers.

Licensed cord blood products

The FDA has licensed several umbilical cord blood products under Biologics License Applications for use in hematopoietic reconstitution. These products are held to manufacturing and testing standards similar to other biological drugs.

Approved cell-based gene therapies

The FDA has approved several cell-based gene therapies — including some chimeric antigen receptor (CAR) T-cell therapies — for specific cancers such as certain B-cell lymphomas, leukemias, and multiple myeloma. These products use a patient's own immune cells that have been genetically modified in a licensed facility. They are approved for narrow, specific indications and are administered in specialized treatment centers.

What is generally not FDA-approved

As of this writing, the FDA has not approved stem cell products for the vast majority of conditions commonly advertised by consumer stem cell clinics, including:

  • Osteoarthritis and joint injuries
  • Multiple sclerosis (outside specific HSCT trials)
  • Amyotrophic lateral sclerosis (ALS)
  • Autism
  • Alzheimer's disease and other dementias
  • Chronic obstructive pulmonary disease (COPD)
  • Erectile dysfunction
  • General "anti-aging" or wellness use

Research is ongoing in many of these areas, but ongoing research is not the same as an approved therapy. For the most current, authoritative list, consult FDA's Cellular & Gene Therapy Products page.