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Melanoma Drug Vindicates Targeted Approach

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Science  18 Dec 2009:
Vol. 326, Issue 5960, pp. 1619
DOI: 10.1126/science.326.5960.1619

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The only metastatic melanoma drug, dacarbazine, was approved by the U.S. Food and Drug Administration in 1975 and has a 15% response rate. So when a phase I trial for a new targeted molecular therapy that binds to and inactivates the BRAF protein, which is mutated in about 60% of melanomas, reported a 70% response rate in September, it shocked the field. Mutant BRAF turns on signaling in a pathway in cells that controls proliferation, and the drug's efficacy validates the theory that targeting this pathway would destroy tumors. But how the drug works remains unclear, and whether other cancer mutations can be practically targeted remains to be seen.

  • * Ken Garber is a freelance writer in Ann Arbor, Michigan.