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

ABSTRACT

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.

KEYWORDS:

• Biliary tract cancer
• cholangiocarcinoma
• gallbladder cancer
• immunotherapy
• immune-checkpoint inhibitors
• nivolumab

Article highlight

• Biliary tract cancer (BTC) comprises a heterogeneous group of aggressive malignancies comprising intrahepatic cholangiocarcinoma, extrahepatic cholangiocarcinoma, gallbladder cancer, and ampulla of Vater cancer.

• Although recent years have witnessed the emergence of novel therapeutic options in BTC patients, medical therapy remains a compelling challenge in these hepatobiliary malignancies.

• Immune checkpoint inhibitors (ICIs) are being investigated in this setting, with controversial results reported so far for ICI monotherapy.

• However, nivolumab has provided interesting antitumor activity in some recently published phase II trials, and the results of ongoing studies are highly awaited.

• In this review, we provide a comprehensive overview of recent studies and available evidence on nivolumab in BTC patients, where this PD-1 inhibitor is in its initial phase of evaluation.

Box 1. Drug summary

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This manuscript was not funded.