Citations: Hyman DM, Puzanov I, Subbiah V, et al. Vemurafenib in multiple nonmelanoma cancers with BRAF V600 mutations. New Engl J Med. 2015;373:726–736.
More than one-third of patients with advanced lung cancer whose tumors had the BRAF V600 mutation responded to the metastatic melanoma drug vemurafenib, according to a recent study in the New England Journal of Medicine.
The first to test vemurafenib in patients with non-melanoma cancers, the trial is considered a ‘basket study’—a new type that explores drug response in patients selected based on specific tumor mutations rather than based on the original sites of the cancer. The BRAF V600 mutation is common in cancers of the skin, but is less common in other cancer types.
“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 senior author José Baselga, MD, PhD, physician-in-chief and chief medical officer at Memorial Sloan Kettering Cancer Center, New York, NY. “While we can—and should—be cautiously optimistic, this is what the future of precision medicine looks like.”
The study included 122 patients with multiple non-melanoma BRAF V600-mutated cancers and two rare conditions involving the overproliferation of white blood cells. In the 20 participants with non-small cell lung cancer, the response rate was 42% and median progression-free survival was 7.3 months, researchers reported. The response rate was 43% in patients with Erdheim-Chester disease and Langerhans cell histiocytosis, according to the study.
Although median treatment duration among those patients was 5.9 months, no one progressed during therapy
Effects were smaller or absent in patients with colorectal cancer, brain tumors, multiple myeloma, anaplastic thyroid cancer, and bile duct cancer.—Jolynn Tumolo
