Drugs used to treat the tumors common in people with a disorder called neurofibromatosis 1 rarely work, and scientists now know why. The chemotherapy drugs target a group of related proteins, call RAS proteins, which are thought to be responsible for these tumors. But researchers at Washington University School of Medicine in St. Louis found that the disease affects only one member of the protein family, and it happens to be the one form of RAS that does not respond well to these particular treatments.
The study, which will appear in the Jan. 1 issue of the journal Cancer Research, suggests where researchers should now look for more promising approaches to treating neurofibromatosis tumors, and may help scientists understand other cancers related to RAS. "The downside is our study proves were not using the right therapies for this particular problem," says principal investigator David H. Gutmann, M.D., Ph.D., the Donald O. Schnuck Family Professor of Neurology and professor of genetics and of pediatrics. "But theres a chance to make lemonade out of this lemon: We now have a rational reason for why these drugs arent working, so we should be able to explore new, more effective treatment options."
About one in 4,000 newborns has neurofibromatosis 1, in which every cell in the body has one normal and one mutated copy of a gene called NF1. If a cells normal copy also is mutated, tumors can form. Children with neurofibromatosis 1 are therefore predisposed to developing a variety of serious complications as they grow older, including skin, spine and brain cancers.
Gila Z. Reckess | EurekAlert!
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