Memorial Sloan Kettering Cancer Center (MSKCC) recently announced results from a “basket study,” an innovative clinical trial design that studies response to drugs based on specific mutations in patients’ tumors rather than where the cancer originated.

The results, published in the New England Journal of Medicine, examined the effects of the drug vemurafenib (Zelboraf) in multiple nonmelanoma BRAFV600 mutated cancers. Vermurafenib has been proven to treat BRAFV600 melanoma. People with ovarian, lung and colorectal cancer were included in the study, as well as those with rare diseases. Before this study, the efficacy of vemurafenib in nonmelanoma cancers was unexamined.

According to Dr. Baselga, the senior author of the study, “This 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.”(

Basket Studies

Basket studies allow the observation of early signals of activity across multiple tumor types simultaneously. This study explored treatment responses among tumors based on their mutation types, and attempted to identify positive signals of activity in individual tumor types that could be earmarked for further study.

One of the advantages of basket studies is the ability to greatly increase the number of patients eligible to receive targeted drugs.

Significant Findings

The findings indicated preliminary clinical efficacy of the drug, vermurafenib, in multiple non-melanoma BRAFV600 mutated cancers. Of the 122 trial participants, clinical activity was observed in various tumor types.

This trial is groundbreaking and the beginning of future studies that focus on cancer-related mutations identified by genomic data.

Importance of Mutational Analysis

The importance of mutational testing and the identification of specific mutations has always been the cornerstone of targeted therapies for GIST.  For more information on the role of specific mutations in GIST, click here.

The study highlights the importance of innovative research, as we begin to explore new solutions that may reach across multiple diseases to provide effective treatment.