Medical technology3 min read
Stem cell therapies
Stem cells can become many types of cells. Their approved medical use today is mainly blood-forming stem cell transplants for blood cancers and some blood disorders. The FDA warns about unapproved “stem cell” products marketed for other conditions, which have caused harms including blindness, tumours and infections.
Stem cell transplantation restores the body's ability to produce blood cells after very high doses of chemotherapy or radiotherapy, and is often used in blood cell cancers such as leukaemia, lymphoma and multiple myeloma, and in other blood disorders including aplastic anaemia and sickle cell disease.
According to the FDA, the only approved stem cell products are blood-forming cells derived from cord blood, for disorders of blood production; no regenerative medicine therapies have been approved for any neurological disorder or any bone or joint condition. Any unapproved product needs FDA oversight in a clinical trial before approval. See Induced pluripotent stem cells.
- In widespread clinical use
- Approved for specific uses
- In clinical trials
- Preclinical
- Concept
Haematopoietic cell transplantation is an established treatment for blood cancers and disorders; other uses are unapproved and need clinical trials.
- Haematopoietic stem cell transplantationApproved for specific uses
Often used in blood cell cancers and some blood disorders.1
- "Regenerative" treatments for joints, nerves and othersIn clinical trials
Not approved for any neurological or bone and joint condition, and need FDA oversight in a clinical trial.2
Status last checked: · What the maturity levels mean
What is it?
Stem cells are cells that can develop into many types of body cells.
Stem cell transplantation is a procedure that restores blood stem cells to people who have lost them because of very high doses of chemotherapy or radiotherapy.
How does it work?
The transplant does not usually act against the cancer directly, but restores the body's ability to produce blood cells; the cells may come from the patient themselves, a donor or an identical twin.
Where is it used today?
Often used in blood cell cancers such as leukaemia, lymphoma and multiple myeloma, and in blood disorders including aplastic anaemia and sickle cell disease.
Limits and risks
Transplantation from a donor may cause graft-versus-host disease, and increases the risk of infection and bleeding.
The FDA has received reports of blindness, tumours, infections and other harms from unapproved stem cell products, and these products are still widely marketed.
No regenerative medicine therapies have been approved for any neurological disorder or any bone or joint condition.
Common questions
A clinic offers "stem cell treatment" for knee pain or neurological diseases; is it approved?
According to the FDA, no regenerative medicine treatment has been approved for bone, joint or neurological conditions, and it has received reports of serious harm from unapproved products. Discuss any such offer with your doctor before any decision.2
Questions for your doctor
- Is stem cell transplantation an option in my case, and where would the cells come from?
- Is the treatment I am being offered approved, or part of a registered clinical trial?
References
- 1National Cancer Institute (NIH). Stem Cell Transplants in Cancer Treatment. www.cancer.gov/about-cancer/treatment/types/stem-cell-transplantHealth agencies & guidelines · Accessed 2026-10-08
- 2U.S. FDA. Important Patient and Consumer Information About Regenerative Medicine Therapies. www.fda.gov/vaccines-blood-biologics/consumers-biologics/important-patient-and-consumer-information-about-regenerative-medicine-therapiesHealth agencies & guidelines · Accessed 2026-10-08
- 3MedlinePlus (NLM). Stem Cells. medlineplus.gov/stemcells.htmlHealth agencies & guidelines · Accessed 2026-10-08
Review status: Edited content · Last updated:
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