Research on Targeted Therapy for Malignant Tumors of the Biliary Tract

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.