FDA warnings about stem cell therapies
Stem cell therapies may offer the potential to treat diseases or conditions for which few treatments exist.
Sometimes called the body’s “master cells,” stem cells are the cells that develop into blood, brain, bones and all of the body’s organs. They have the potential to repair, restore, replace and regenerate cells, and could possibly be used to treat many medical conditions and diseases.
Today, doctors routinely use stem cells that come from bone marrow or blood in transplant procedures to treat patients with cancer and disorders of the blood and immune system.
But the U.S. Food and Drug Administration is concerned that some patients seeking cures and remedies are vulnerable to stem cell treatments that are illegal and potentially harmful.
FDA regulates only U.S. use
The FDA has the authority to regulate stem cell products in the United States.
The only stem cell-based products that are FDA-approved for use in the United States consist of blood-forming stem cells (hematopoietic progenitor cells) derived from cord blood.
These products are approved for limited use in patients with disorders that affect the body system that is involved in the production of blood (called the hematopoietic system).
These FDA-approved stem cell products are listed on the FDA website. Bone marrow also is used for these treatments but is generally not regulated by the FDA for this use.
With limited exceptions, new products being investigated must go through a thorough FDA review process as investigators prepare to determine the safety and effectiveness of products in well-controlled human studies, called clinical trials. The FDA has reviewed many stem cell products for use in these studies. (You can learn more about clinical trials on the FDA’s website.)
That said, some clinics may inappropriately advertise stem cell clinical trials without submitting to the FDA’s review process. Some clinics also may falsely advertise that FDA review and approval of the stem cell therapy is unnecessary.
All medical treatments have benefits and risks. But unproven stem cell therapies can be particularly unsafe.
For instance, attendees at a 2016 FDA public workshop discussed several cases of severe adverse events. One patient became blind due to an injection of stem cells into the eye. Another patient received a spinal cord injection that caused the growth of a spinal tumor.
Other potential safety concerns for unproven treatments include:
- Administration site reactions,
- The ability of cells to move from placement sites and change into inappropriate cell types or multiply,
- Failure of cells to work as expected, and
- The growth of tumors.
Note: Even if stem cells are your own cells, there are still safety risks such as those noted above. In addition, if cells are manipulated after removal, there is a risk of contamination of the cells.
Before trying stem cell therapies
You may be told that because these are your cells, the FDA does not need to review or approve the treatment. That is not true [in the United States].
If you’re considering treatment in the United States:
- Ask if the FDA has reviewed the treatment. Ask your healthcare provider to confirm this information. Ask for this information before getting treatment — even if the stem cells are your own.
- Request the facts and ask questions if you don’t understand. Make sure you understand the entire process and known risks before you sign.
If you’re considering treatment in another country:
- Learn about regulations that cover products in that country.
- Know that the FDA does not have oversight of treatments done in other countries. The FDA typically has little information about foreign establishments or their stem cell products.
- Be cautious. If you’re considering a stem cell-based product in a country that may not require regulatory review of clinical studies, it may be hard to know if the experimental treatment is reasonably safe.