下一代肿瘤不可知靶点即将出现
Next-generation tumor-agnostic targets on the horizon.
肿瘤不可知药物开发将肿瘤学重新聚焦于共享的分子依赖性而非组织来源，从而能够针对跨肿瘤的罕见可操作驱动因素进行高效开发。
99.3分
★★★★★
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
胆道恶性肿瘤靶向治疗研究
英文原题：Research on Targeted Therapy for Malignant Tumors of the Biliary Tract.
研究概要
胆道癌（BTCs）是侵袭性恶性肿瘤，发病率不断上升，预后极差。
中文摘要
胆道癌（BTCs）是一种侵袭性恶性肿瘤，发病率不断上升，预后极差。本综述综合了精准肿瘤学和免疫治疗领域的变革性进展，这些进展正在重塑BTC的管理格局。基因组分析揭示了可靶向的变异——IDH1/2突变、FGFR2融合、HER2异常、BRAF V600E——推动了特定抑制剂（ivosidenib、FGFR抑制剂、HER2靶向ADC/抗体、dabrafenib/trametinib）的临床成功。这些药物在分子定义的亚组中展现出前所未有的缓解率和生存获益，优于化疗。新兴的免疫疗法，包括检查点阻断（尤其是针对MSI-H/dMMR肿瘤）、过继性细胞治疗和癌症疫苗，显示出良好前景，且常与靶向方法产生协同作用。然而，克服肿瘤异质性、耐药机制以及优化联合策略仍是关键挑战。这种向分子指导治疗的模式转变，为改善BTC患者生存带来了重大希望。
展开英文摘要原文
Biliary tract cancers (BTCs) are aggressive malignancies with rising incidence and dismal outcomes. This review synthesizes transformative advances in precision oncology and immunotherapy reshaping BTC management. Genomic profiling reveals targetable alterations-IDH1/2 mutations, FGFR2 fusions, HER2 aberrations, BRAF V600E-driving the clinical success of specific inhibitors (ivosidenib, FGFR inhibitors, HER2-targeted ADCs/antibodies, dabrafenib/trametinib). These agents demonstrate unprecedented response rates and survival benefits in molecularly defined subsets compared to chemotherapy. Emerging immunotherapies, including checkpoint blockade (especially for MSI-H/dMMR tumors), adoptive cell therapy, and cancer vaccines, show promise, often synergizing with targeted approaches. However, overcoming tumor heterogeneity, resistance mechanisms, and optimizing combination strategies remain critical challenges. This paradigm shift towards molecularly guided therapies offers significant hope for improving BTC patient survival.
论文信息
- 作者
- Yang Y、Zhong H、Zhang J、Wang P
- 单位
- Department of General Surgery, Affiliated Hospital of Nantong University, Nantong, Jiangsu, 226001, People's Republic of China.China
- 文献类型
- 综述
- 期刊
- OncoTargets and therapy2026
- 原文标识
- PubMed 42146765 · DOI 10.2147/OTT.S593457