New Cancer Drug Vermurfenib Treats BRAF V600 Mutation in Both Melanoma and Nonmelanoma

The BRAF V600 mutation occurs in almost 50% of melanomas and results in activation of a MAPK pathway. Vemurafenib, a new cancer, is an inhibitor of the BRAF V600 kinase and is associated with a 50% response rate and improved survival in patients with the BRAF V600 mutation who also have metastatic melanoma. The efficacy of vermurafenib in nonmelanoma cancers has not been heavily studied. Thus to treat nonmelanoma cancer patients with the BRAF V600 gene, Hyman and his colleagues designed a study to test the efficacy of vermurafenib in other various types of cancer.


The study included patients with the following nonmelanoma cancers: non-small-cell lung cancer, ovarian cancer, colorectal cancer, cholangiocarcinoma, and breast cancer. The primary outcomes was to measure the response rate of the drug, progression of the tumor, and overall survival rate. In the group with the non-small-cell lung cancer, the response rate was 42% and the survival rate extended 7.3 months. There were positive responses in the drug treating the other variations of cancer, but this was the most significant one. Therefore the BRAF V600 gene is a good target gene for some nonmelanoma cancers, especially non-small-cell lung cancer. In addition, vemurafenib is a drug that can treat both melanoma and nonmelanoma cancer.


It is always encouraging when a new cancer drug is developed and proven to be effective. What is so remarkable about this drug is that although it treats a very specific gene mutation, it treats a significant amount of different outcomes from this mutation. Non-small cell lung cancer is a type of lung cancer that causes death in 50% of its patients so finding a drug that can prolong life and decrease tumor growth is a great accomplishment.


Hyman D, Puzannov I, Subbiah V, et al. Vermurfenib in multiple nonmelanoma cancers with BRAFV600 Mutations. N Engl J Med. 2015; 373:726-736.