精准肿瘤学中组织不可知治疗方法的不断发展。

The evolving landscape of tissue-agnostic therapies in precision oncology.

机构信息

Sarah Cannon Research Institute, Nashville, Tennessee, USA.

Department of Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.

出版信息

CA Cancer J Clin. 2024 Sep-Oct;74(5):433-452. doi: 10.3322/caac.21844. Epub 2024 May 30.

Abstract

Tumor-agnostic therapies represent a paradigm shift in oncology by altering the traditional means of characterizing tumors based on their origin or location. Instead, they zero in on specific genetic anomalies responsible for fueling malignant growth. The watershed moment for tumor-agnostic therapies arrived in 2017, with the US Food and Drug Administration's historic approval of pembrolizumab, an immune checkpoint inhibitor. This milestone marked the marriage of genomics and immunology fields, as an immunotherapeutic agent gained approval based on genomic biomarkers, specifically, microsatellite instability-high or mismatch repair deficiency (dMMR). Subsequently, the approval of NTRK inhibitors, designed to combat NTRK gene fusions prevalent in various tumor types, including pediatric cancers and adult solid tumors, further underscored the potential of tumor-agnostic therapies. The US Food and Drug Administration approvals of targeted therapies (BRAF V600E, RET fusion), immunotherapies (tumor mutational burden ≥10 mutations per megabase, dMMR) and an antibody-drug conjugate (Her2-positive-immunohistochemistry 3+ expression) with pan-cancer efficacy have continued, offering newfound hope to patients grappling with advanced solid tumors that harbor particular biomarkers. In this comprehensive review, the authors delve into the expansive landscape of tissue-agnostic targets and drugs, shedding light on the rationale underpinning this approach, the hurdles it faces, presently approved therapies, voices from the patient advocacy perspective, and the tantalizing prospects on the horizon. This is a welcome advance in oncology that transcends the boundaries of histology and location to provide personalized options.

摘要

肿瘤不可知疗法代表了肿瘤学的范式转变,它改变了传统上基于肿瘤起源或位置来描述肿瘤的方法。相反,它专注于导致恶性生长的特定遗传异常。肿瘤不可知疗法的分水岭时刻出现在 2017 年,美国食品和药物管理局历史性地批准了 pembrolizumab,一种免疫检查点抑制剂。这一里程碑标志着基因组学和免疫学领域的结合,因为一种免疫治疗药物基于基因组生物标志物(微卫星不稳定性高或错配修复缺陷(dMMR))获得批准。随后,批准了 NTRK 抑制剂,旨在针对各种肿瘤类型中常见的 NTRK 基因融合(包括儿科癌症和成人实体瘤),进一步突显了肿瘤不可知疗法的潜力。美国食品和药物管理局继续批准针对特定靶点的靶向治疗(BRAF V600E、RET 融合)、免疫治疗(肿瘤突变负担≥10 个突变/兆碱基、dMMR)和抗体药物偶联物(Her2 阳性-免疫组化 3+表达)具有泛癌疗效,为患有特定生物标志物的晚期实体瘤的患者带来了新的希望。在这篇全面的综述中,作者深入探讨了组织不可知靶点和药物的广阔领域,阐明了这种方法的基本原理、面临的障碍、目前批准的治疗方法、患者倡导观点的声音以及未来的诱人前景。这是肿瘤学的一个可喜进展,超越了组织学和位置的界限,提供了个性化的选择。

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索