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Fred Hutchinson Cancer Research Center study shows retinoids kill childhood brain tumor cells

Researchers find that vitamin A derivatives may be highly effective and minimally toxic treatments for medulloblastoma, the most common form of childhood brain cancer. Clinical trials of the drugs, known as retinoids, are being planned for children who are at high risk for tumor relapse following standard therapy.

A study led by Fred Hutchinson researchers, Drs. Andrew Hallahan and James Olson showed that retinoids killed cancer cells from medulloblastoma tumors that had been surgically removed from patients as well as tumors that had been grafted onto mice. Through genome analysis, the scientists also identified a protein in medulloblastoma cells that is triggered by retinoids to initiate cell death, a finding that is likely to lead to the development of additional therapies for the disease. The study appears in the Aug. 3 issue of Nature Medicine.

Because retinoids already have received approval from the Food and Drug Administration for treatment of another childhood cancer, neuroblastoma, researchers expect that the drugs will enter clinical trials in pediatric medulloblastoma patients with minimal delay.

Medulloblastomas arise from primitive cells in the back of the brain, or cerebellum, a region important for motor control and spatial orientation. The disease primarily strikes children under the age of 7. Standard therapy, which includes surgical removal of the tumor followed by radiation and a year of chemotherapy, offers about a 70 percent chance of survival for children over age 3 who do not have recurrent cancer.

High-risk children, who include those under age 3 or who have recurrent disease, have a much lower chance of survival with standard therapy. For that reason-and because of the toxic side effects of radiation and chemotherapy in young children-scientists are eager to find new treatments.

According to Hallahan, retinoids could prove to be even more effective at treating medulloblastoma than neuroblastoma.
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Contact: Susan Edmonds
sedmonds@fhcrc.org
206-667-2896
Fred Hutchinson Cancer Research Center
4-Aug-2003


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