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Established Hematopoietic Stem-Cell Transplantation: Context

Blood and bone marrow stem-cell transplants are established medical care for certain cancers and blood disorders. This page explains what they are, their risks, and why they do not validate clinic offers.

Content last updated . This page has not been medically reviewed.

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Quick answer
Hematopoietic (blood-forming) stem-cell transplantation is the long-established, best-evidenced use of stem cells in medicine. It is carried out in specialist hospitals for specific diseases and carries serious risks. It is also often used by marketers as proof that "stem cell therapy works" for unrelated conditions, which it does not show. Information only, not medical advice.

What it is

Bone marrow is the spongy tissue inside some bones; it contains immature cells that can develop into red blood cells, white blood cells and platelets. [1] A transplant replaces a person's faulty blood-forming stem cells. [1]

The National Cancer Institute describes stem cell transplants as procedures that restore blood stem cells in people whose own were destroyed by the high doses of chemotherapy or radiation used to treat certain cancers, blood disorders and autoimmune disorders. [2] The cells can come from the bloodstream, bone marrow or umbilical cord blood. [2]

Evidence label: established for the conditions below, in the settings where doctors use it.

Who it is used for

MedlinePlus lists leukemia, multiple myeloma, severe blood diseases such as thalassemias, aplastic anemia and sickle cell anemia, and certain immune deficiency diseases. [1] NCI says transplants are most often used for cancers affecting blood cells, such as leukemia, lymphoma, multiple myeloma and myelodysplastic syndromes, and that transplants for other cancer types are being studied in clinical trials. [2]

How it mostly works

NCI explains that transplants usually do not attack cancer directly. They restore the body's ability to make blood cells after very high-dose treatment. In leukemia, donor cells may also attack remaining cancer cells, called the graft-versus-leukemia effect. [2]

Types and matching

Transplants can be autologous (your own cells), allogeneic (a donor's cells) or syngeneic (an identical twin's cells). [2] Donors are matched by human leukocyte antigens (HLAs); the more you share, the better the chance your body accepts the cells. [2] See autologous vs allogeneic cell products.

Why this does not validate stem cell clinics

A transplant of blood-forming cells for a blood disease says nothing about injecting other cell products into joints, the brain or the eye. See cell-therapy clinical trials vs commercial treatments and mesenchymal stromal cell research. For the field overall, see stem cells explained.

Frequently asked questions

Is a bone marrow transplant the same as a stem cell transplant?

They overlap. NCI says transplants from bone marrow may be called bone marrow transplants, from the bloodstream peripheral blood stem cell transplants, and from cord blood cord blood transplants. [2]

Is a transplant an alternative medicine?

No. It is an established hospital procedure for specific diseases, with serious risks. [1]

What is graft-versus-host disease?

It occurs when donor white blood cells see the recipient's cells as foreign and attack them, damaging skin, liver, intestines and other organs. [2]

Does this prove stem cell injections work for arthritis or other conditions?

No. FDA says regenerative medicine therapies have not been approved for orthopedic conditions such as osteoarthritis. [7]

Who decides if I need a transplant?

Your specialist team weighs your disease, donor availability and other health problems. [2]

More details: evidence, limits and cautions

The details behind this page: cautions, limits of the evidence and where sources disagree. Open any note to read it.

Risks
MedlinePlus states that bone marrow transplantation has serious risks and that some complications can be life-threatening. [1] NCI lists short-term problems such as nausea and fatigue, long-term problems such as infertility, cataracts, secondary cancers and organ damage, and graft-versus-host disease, in which donor white blood cells attack the recipient's tissues. [2] Graft-versus-host disease is one reason regulators have approved cell products for it. See the next section.
Regulatory context (United States)
FDA's approved list, read 2026-10-22, includes several cord blood products, and Omisirge. [3] FDA's product page lists Omisirge for patients 12 and older with blood cancers planned for cord blood transplantation to shorten neutrophil recovery and reduce infection, and for patients 6 and older with severe aplastic anemia after reduced-intensity conditioning. [4] FDA approved Ryoncil, a mesenchymal stromal cell therapy, for children 2 months and older with steroid-refractory acute graft versus host disease after transplant. [5]

FDA consumer pages say that the stem cell products approved for blood-forming indications do not extend to other uses. Its consumer alert states they are approved for disorders of blood production and not for other uses. [6] That page was published 2020 and listed as current as of 2024, so check the newer approved list above. [6]

References
  1. MedlinePlus. Bone Marrow Transplantation. Read 2026-10-22.
  2. National Cancer Institute. Stem Cell Transplants in Cancer Treatment. Read 2026-10-22.
  3. U.S. Food and Drug Administration. Approved Cellular and Gene Therapy Products. Content current as of 09/17/2026. Read 2026-10-22.
  4. U.S. Food and Drug Administration. OMISIRGE. Read 2026-10-22.
  5. U.S. Food and Drug Administration. FDA approves remestemcel-L-rknd for steroid-refractory acute graft versus host disease in pediatric patients. Read 2026-10-22.
  6. U.S. Food and Drug Administration. Consumer Alert on Regenerative Medicine Products Including Stem Cells and Exosomes. July 22, 2020; content current as of 04/09/2024. Read 2026-10-22.
  7. U.S. Food and Drug Administration. Important Patient and Consumer Information About Regenerative Medicine Therapies. Content current as of 04/08/2024. Read 2026-10-22.
Education, not medical advice. This page is for general information. It does not diagnose or treat any condition and is not a substitute for care from a qualified health professional. Natural does not automatically mean safe. Ask a clinician or pharmacist before changing your care, especially if you take medication, are pregnant or breastfeeding, or have a health condition.