Advances in target drugs and immunotherapy for biliary tract cancer.

IF 3.8 3区医学 Q2 GASTROENTEROLOGY & HEPATOLOGY

Expert Review of Gastroenterology & Hepatology Pub Date : 2024-10-01 Epub Date: 2024-11-15 DOI:10.1080/17474124.2024.2416230

Chiara Deiana, Chiara Ricci, Mahrou Vahabi, Mahsoem Ali, Giovanni Brandi, Elisa Giovannetti

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引用次数: 0

Abstract

Introduction: After years of treatment stagnation in biliary tract cancers (BTC), there has been a notable shift with the emergence of targeted therapies and immunotherapy, leading to substantial progress in tackling this aggressive disease.

Areas covered: We provide a comprehensive overview of the target therapies that are already part of the treatment algorithm for BTC, such as FGFR, IDH, and HER2 inhibitors. Additionally, we delve into some less known targets that are being explored, such as KRAS proto-oncogene, MAPK cascade, PI3K/AKT/mTOR pathway and novel molecules directed against P53, claudin, histones, and mitochondrial metabolism. Furthermore, we discuss agnostic drugs and analyze the efficacy data available for BTC specifically. We also examine the expanding world of immunotherapy, with an eye on predictive factors of response for immune checkpoint inhibitors, and on novel immune drugs such as chimeric antigen receptor (CAR)-T and vaccines.

Expert opinion: In the expert opinion, we discuss the problem of the scarcity of patients eligible for target therapies and how can clinical trials be designed to overcome this challenge. We also summarize the most promising trials that have the potential to change clinical practice both for immunotherapies and target drugs.

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胆道癌靶向药物和免疫疗法的进展。

简介：在胆道癌（BTC）的治疗停滞多年之后，靶向疗法和免疫疗法的出现带来了显著的转变，使这一侵袭性疾病的治疗取得了重大进展：我们全面概述了已成为 BTC 治疗算法一部分的靶向疗法，如表皮生长因子受体（FGFR）、IDH 和 HER2 抑制剂。此外，我们还深入探讨了一些鲜为人知的靶点，如 KRAS 原癌基因、MAPK 级联、PI3K/AKT/mTOR 通路以及针对 P53、Claudin、组蛋白和线粒体代谢的新型分子。此外，我们还讨论了不可知药物，并分析了专门针对 BTC 的疗效数据。我们还审视了不断扩展的免疫疗法领域，关注免疫检查点抑制剂的反应预测因素，以及嵌合抗原受体（CAR）-T 和疫苗等新型免疫药物：在专家意见中，我们讨论了符合靶向治疗条件的患者稀缺的问题，以及如何设计临床试验来克服这一挑战。我们还总结了最有前景的试验，这些试验有可能改变免疫疗法和靶向药物的临床实践。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Expert Review of Gastroenterology & Hepatology GASTROENTEROLOGY & HEPATOLOGY-

CiteScore

6.80

自引率

2.60%

发文量

审稿时长

6-12 weeks

期刊介绍： The enormous health and economic burden of gastrointestinal disease worldwide warrants a sharp focus on the etiology, epidemiology, prevention, diagnosis, treatment and development of new therapies. By the end of the last century we had seen enormous advances, both in technologies to visualize disease and in curative therapies in areas such as gastric ulcer, with the advent first of the H2-antagonists and then the proton pump inhibitors - clear examples of how advances in medicine can massively benefit the patient. Nevertheless, specialists face ongoing challenges from a wide array of diseases of diverse etiology.