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Cancer Overview

Landmark Cancer “Basket Study”

Researchers from Memorial Sloan Kettering Cancer Center 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 where their cancers originated.

Published in August 2015 in the New England Journal of Medicine, the early phase II study, led by Physician-in-Chief and Chief Medical Officer José Baselga, MD, PhD, looked at the effect of vemurafenib (Zelboraf®) in multiple nonmelanoma BRAFV600-mutated cancers in 122 patients from 23 centers around the world. 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 nonmelanoma cancers has remained unexplored despite significant therapeutic potential.

A release from Sloan Kettering quotes Dr. Baselga, the study’s senior author, as saying, “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. 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. The first to follow this model, this new study aims to explore treatment responses among tumors based on their mutation types and to identify promising signals of activity in individual tumor types that could be pursued in subsequent studies. The results will ultimately guide researchers in looking for different drug targets or developing therapies that combine vemurafenib with complementary treatments.

Basket studies also have the ability to greatly increase the number of patients eligible to receive certain drugs. The mixed efficacy seen in this study proves that drugs can reach patients beyond the current approved use but, expectedly, do not work for everyone. As a pioneering trial, this data demonstrates the promising benefits of basket studies and the need for more work to be done with these types of trials.