DRUG TARGETS, BRAF: A basket study (n=172) of vemurafenib in assorted BRAF V600-mutant tumours reported ORR 33%, with responses seen in 13 different tumour histologies.

Kevin Sovet

May 18, 2020

SCIENTIFIC

DRUG TARGETS, BRAF

A basket study (n=172) of vemurafenib in assorted BRAF V600-mutant tumours reported ORR 33%, with responses seen in 13 different tumour histologies.

 

Pan-Cancer Efficacy of Vemurafenib in BRAFV600-Mutant Non-Melanoma Cancer

DOI: 10.1158/2159-8290.CD-19-1265

 

Abstract

BRAF V600 mutations occur in a wide range of tumor types and RAF inhibition has become standard in several of these cancers. Despite this progress, BRAF V600 mutations have historically been considered a clear demonstration of tumor lineage context-dependent oncogene addiction, based predominantly on the insensitivity of RAF inhibition in colorectal cancer. However, the true broader activity of RAF inhibition pan-cancer remains incompletely understood. To address this, we conducted a multi-cohort 'basket' study of the BRAF inhibitor vemurafenib in non-melanoma BRAF V600 mutation-positive solid tumors. In total, 172 patients with 26 unique cancer types were treated, achieving an overall response rate of 33% and median duration of response of 13 months. Responses were observed in 13 unique cancer types, including historically treatment-refractory tumors such as cholangiocarcinoma, sarcoma, glioma, neuroendocrine carcinoma, and salivary gland carcinomas. Collectively, these data demonstrate that single-agent BRAF inhibition has broader clinical activity than previously recognized.

More info : https://cancerdiscovery.aacrjournals.org/content/early/2020/02/06/2159-8290.CD-19-1265