Inhibitor of MEK1/2, selumetinib, for biliary tract cancer

Abstract

It is necessary to establish effective chemotherapy to improve the survival of patients with biliary tract cancer. Although the useful of some molecular-targeted agents as first-line therapies has been investigated, none have been found to exert satisfactory efficacy. In this article, we report the results of a Phase II study of selumetinib in patients with metastatic biliary cancer. Selumetinib is an inhibitor of MEK1/2 targeting the RAS/RAF/MEK/extracellular signal-related kinase pathway. Three out of 28 patients showed a confirmed partial response, representing a response rate of 12%. The median progression-free survival was 3.7 months and the median overall survival was 9.8 months. The most common toxicities were rash, xerostomia and nausea. Most toxicities were grade 1 or 2, and the most common grade 3/4 toxicities were diarrhea and nausea. All toxicities were manageable and reversible. The results warrant further evaluation of the use of selumetinib in patients with metastatic biliary cancer.