Sloan-Kettering Reports First “Basket” Study, Categorizing Specific Cancer Mutations and Responses to Targeted Drugs, Exclusive of Cancer Origin Site; Described As “First Deliverable of Precision Medicine”

Researchers from the Memorial Sloan Kettering Cancer Center (MSKCC) in New York City have announced results from the first published “basket” study, a new form of clinical trial design that explores responses to drugs based on the specific mutations in patients' tumors rather than the site where their cancer originated. Published in today’s (August 20, 2015 issue of the New England Journal of Medicine, the early phase II study, led by MSKCC Physician-in-Chief and Chief Medical Officer José Baselga, M.D., Ph.D., looked at the effect of vemurafenib (Zelboraf®) in multiple non-melanoma BRAFV600-mutated cancers in 122 patients from 23 centers around the world. The article is titled “Vemurafenib in Multiple Nonmelanoma Cancers with BRAFV600 Mutations.” “Vemurafenib previously has been proven to treat BRAFV600-mutated melanoma. People with lung, colorectal, and ovarian cancers were among those included in the study, as well as people with rare diseases, such as Erdheim-Chester disease. Until this point, the efficacy of vemurafenib in non-melanoma cancers has remained unexplored despite significant therapeutic potential. "This study is the first deliverable of precision medicine. We have proven that histology-independent, biomarker-selected basket studies are feasible and can serve as a tool for developing molecularly targeted cancer therapy," said Dr. Baselga, the study's senior author. "While we can -- and should -- be cautiously optimistic, this is what the future of precision medicine looks like." Basket studies permit the detection of early signals of activity across multiple tumor types simultaneously, while allowing for the possibility that tumor lineage might influence drug sensitivity.
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