The National Cancer Institute's NCI-MATCH trial will be a massive precision medicine experiment at more than 2,400 sites around the country.
About 3,000 patients will have their tumor genes sequenced to see what mutations or pathways fuel their disease. About 1,000 patients whose tumor characteristics most closely match one of the 20 or so gene-targeting drugs offered in the study will be put into groups of about 30 patients to get that drug.
Many cancers share the same gene mutations and so a drug that targets one of these for a specific cancer, such as breast, may work against other types, such as lung. "We're hoping that a substantial minority of the patients that are tested will actually have rare or uncommon cancers" so more can be learned about what genes fuel them, Lowy said.
Dr. Richard Pazdur, cancer drugs chief at the Food and Drug Administration, warned that although everyone hopes that targeting drugs to gene mutations will improve survival, "this may be far more complex than we realize."
The FDA has never approved a drug that was not aimed at a specific tumor type such as breast cancer, but if a drug shows promise for a particular pathway involved in many tumor types, it could be approved for that use, he said.
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