Nivolumab: an investigational agent for the treatment of biliary tract cancer

Introduction: Biliary tract cancers (BTCs) include four uncommon primary biliary malignancies characterized by poor prognosis: intrahepatic cholangiocarcinoma, extrahepatic cholangiocarcinoma, gallbladder cancer, and ampulla of Vater cancer. To date, the available therapeutic options are limited; nevertheless, with the advent of targeted therapy and immunotherapy, the treatment scenario is constantly changing, opening toward future possibilities for the personalized treatment of BTC. Among these treatments, nivolumab is currently being investigated in BTC. Areas covered: This review provides a comprehensive overview of the recent progress and future perspectives of immunotherapy for the treatment of BTC, with a particular focus on nivolumab, which has demonstrated its effectiveness in many malignancies and is in its initial phase of evaluation in BTC. Expert opinion: BTCs are not among those tumors that demonstrated remarkable sensitivity to immunotherapy, such as melanoma or non-small-cell lung cancer. However, the results obtained with nivolumab are not disappointing if we analyze the population of the trials testing its efficacy, including pretreated patients, unselected for PD-L1 expression and with different BTCs. In our opinion, ICIs such as nivolumab could fill an empty spot in the treatment of selected BTC patients, especially in combination with other agents, and the results of ongoing clinical trials are awaited.