Skip to content

Stem Cell Frequently Asked Questions


What is stem cell therapy?

A stem cell therapy is a treatment that uses stem cells, or cells that come from stem cells, to replace or to repair an individual’s cells or tissues that are damaged. The stem cells might be put into the blood, or transplanted into the damaged tissue directly, or even recruited from the individual’s own tissues for self-repair.

For what diseases or conditions are stem cell treatments well established?

The range of diseases for which there are proven treatments based on stem cells is still extremely small. Disorders of the blood and immune system and acquired loss of bone marrow function can, in some cases, be treated effectively with blood stem cell transplantation.

Doctors have been transferring blood stem cells by bone marrow transplant for more than 50 years, and advanced techniques for collecting blood stem cells are now used clinically. Umbilical cord blood, like bone marrow, is often collected as a source of blood stem cells and is being used experimentally as an alternative to bone marrow in transplantation.

Other tissue-specific stem cells may also play a role in tissue transplants that have been performed for several years. For tissues and organs such as skin and cornea, stem cells contained in these tissues contribute to long-term regeneration. Other stem cell treatments are still experimental, including spinal cord injury. This means that it has not yet been shown that this treatment is safe or that it will work.

What are some of the special considerations for stem cell therapies?

Therapies based on stem cells are largely new and there is a lot that we still need to learn.

To start, there are particular challenges in preparing stem cells for use as a medicine. Unlike drugs, stem cells cannot necessarily be produced and tested for quality in large batches, and treatments may even be specific to one individual. For most diseases, it is still being determined which cells will work best to repair a particular damaged or diseased tissue, and how to get those cells to the right place in the body.

Furthermore, side effects and long-term safety must be determined, since transplanted cells may remain for many years in peoples’ bodies. Therefore, careful monitoring and extended follow-up of individuals who receive stem cell treatments is extremely important.

What are the differences between an approved clinical treatment and an experimental intervention?

An approved clinical treatment is a medical practice that has been shown through a formal process of clinical trials to be reasonably safe and effective for treating a particular disease or condition. Normally, such treatments will be approved by a national or regional regulatory agency, for example, the US Food and Drug Administration (FDA) or the European Medicines Agency (EMEA).

An experimental intervention is new, untested, or different from the usual medical treatment. It has not yet been proven that it is safe or that it will work in treating the particular disease. Hence, you should never have to pay for an experimental intervention because you are exposing yourself to unknown risk.

How do I know if an approved stem cell therapy is safe?

No medical treatment can ever be described as completely safe. There are risks involved with all medical treatment, some small, some large. These risks, even if they are small, should be explained clearly to you by a medical professional. Furthermore, the known benefits should outweigh the risks.

What should I look for if I am considering a stem cell therapy?

You need to be sure that there is good scientific evidence that the treatment is safe and effective, and that your rights are being respected. To begin, ask for evidence that:

  • Preclinical studies have been published, and reviewed and repeated by other experts in the field.
  • The providers have approval from an independent committee such as an Institutional Review Board (IRB) or Ethics Review Board (ERB) to make sure the risks are as low as possible and are worth any potential benefits, and that your rights are being protected.