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Stem Cell Clinical Trials: 11 Key Areas

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



  • Goal: Neural stem cells are being used in clinical trials that aim to destroy glioblastoma, a type of brain cancer, when surgery isn't an option.
  • What's being done: The City of Hope, a medical center in California, is genetically modifying neural stem cells to make an enzyme that converts a nontoxic drug (5-Fluorocytosine or 5-FC) into an cancer drug (5-Fluorouracil or 5-FU). The researchers inject the modified neural stem cells into the patient's brain, hoping that the stem cells will travel to the tumor and latch onto it. Then the patients get 5-FC. When 5-FC reaches the tumor site, the attached stem cells help convert it to the cancer drug, 5-FU. The goal is to shrink or destroy the glioblastoma, while sparing the rest of the body from the toxic effects.
  • Does it work? The trial, the first to test this treatment in people, is still under way, so it's too soon to know if it's safe and effective.

Leukemia and Other Blood Cancers and Disorders:

One of the original uses of stem cells (from bone marrow and umbilical cord blood) is to treat blood and immune disorders. Bone marrow or cord blood transplant has become standard treatment for some of these conditions.

The National Bone Marrow Donor Program web site has a list of diseases that can now be treated with hematopoietic (blood-forming) stem cells. These include various leukemias and lymphomas.

Cartilage Repair

  • Goal: Use stem cells to make new cartilage.
  • What's being done: There haven't been many trials in people yet. Some researchers have reported using patients' own adult stem cells (typically taken from their bone marrow), embedding those stem cells into a gel or onto a collagen sheet, and placing it onto the area of cartilage damage (such as the knee or ankle).
  • Does it work? There haven't been enough studies to tell. So far, the results are mixed because the tissue made by the stem cells seems to vary in its quality and durability, according to a 2011 review published in the Open Orthopaedics Journal.
Reviewed on July 11, 2012

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