J Dig Cancer Res.  2024 Dec;12(3):176-183. 10.52927/jdcr.2024.12.3.176.

Recent Advances in Immunotherapy and Targeted Therapy for Biliary Tract Cancer

• Lee JW
• Kang H

• KMID: 2563742
• DOI: http://doi.org/10.52927/jdcr.2024.12.3.176

Abstract

Biliary tract cancer (BTC) is a rare malignancy with increasing incidence and mortality over the past two decades. Despite its unique anatomical and molecular features, BTC is typically diagnosed at advanced stages, thereby limiting treatment options and resulting in poor prognosis. Recent advances in molecular profiling and immunotherapy have revolutionized the BTC management, illustrating its molecular and immunologic underpinnings. Immune checkpoint inhibitors such as durvalumab and pembrolizumab, when combined with gemcitabine/ cisplatin, provide considerable survival benefits, with phase 3 trials such as TOPAZ-1 and KEYNOTE-966 demonstrating improved overall and progression-free survivals with immune checkpoint inhibitors compared to the conventional gemcitabine/cisplatin therapy. Therapies targeting FGFR2 fusions, IDH1 mutations, and HER2 overexpression have further expanded the therapeutic landscape, offering precision medicine opportunities for patients with actionable mutations. However, challenges persist owing to the rarity of BTC and inherent limitations of small cohort studies. Future research should focus on large, multicenter, prospective trials, establish new treatment paradigms, and expand therapeutic applications. In addition, integrating next-generation sequencing into routine practice will enhance molecular-based treatment approaches. This review explores the latest advancements in BTC immunotherapy and targeted therapies, emphasizing their clinical applicability in improving patient outcomes.

Keyword

Biliary tract neoplasms; Immunotherapy; Molecular targeted therapy; Immune checkpoint inhibitors; Precision medicine