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Stem Cell Clinical Trials: 11 Key Areas

What's happening as stem cell treatments are tested in people.
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Parkinson's Disease

Two clinical trials of stem cell treatments are listed on the National Institutes of Health's clinical trials web site. One of those trials is in China, using stem cells from patients' own bone marrow. The other trial, listed as taking place in Mexico, uses stem cells from patients' fat. Both trials are very small (20 patients for the Chinese trial and 10 for the one in Mexico). It is far too early to know if either approach will work.

Alzheimer's Disease

Stem cell research has been done in mice but not in people with Alzheimer's disease.

ALS (Lou Gehrig's Disease)

  • Goal: Test that safety of delivering embryonic stem cells to the spinal cord.
  • What's being done: This trial is being conducted at Emory University and is led by the University of Michigan's Eva Feldman, MD.
  • Does it work? So far, three patients have gotten the stem cell procedure. No side effects were seen, so the FDA has approved them getting a second treatment, higher up in the spinal cord. This trial is not designed to see if the procedure improves their ALS -- just to see if it's safe.

Multiple Sclerosis

  • Goal: Use stem cells to suppress and then reset the immune system to work without MS.
  • What's being done: Clinical trials involve suppressing a multiple sclerosis patient's immune system, then transplanting adult stem cells to rebuild the immune system -- without MS. The stem cells used are those that make blood and are typically found in the bone marrow or umbilical cord blood.
  • Does it work? It's too soon to know. However, an Italian study shows some success. Researchers at Italy's University of Genoa studied 74 MS patients. First, their immune systems were suppressed. Then they got transplants of their own blood-forming (hematopoietic) stem cells. Two patients died from "transplant-related causes," the researchers report. After five years, 66% of patients had remained stable or improved. The study concluded that the therapy "has a sustained effect in suppressing disease progression in aggressive MS cases unresponsive to conventional therapies" and "can also cause a sustained clinical improvement," especially in people with the relapsing-remitting form of MS.

Caution: Because the immune system has to be suppressed before stem cell treatment, "the benefits need to substantially outweigh the risk," states a review of stem cell treatment trials published in BMC Medicine.

Stem cell clinical trials are also being done for other autoimmune diseases, including lupus, Crohn's disease, and rheumatoid arthritis, according to the review published in BMC Medicine. It's not yet clear how well those treatments work.

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