Research advances in molecular targeted therapy for advanced biliary tract cancer

Molecular targeted therapy has become a new hot spot with the in- depth basic research on advanced biliary tract cancer (ABTC) . Phase Ⅱ and Ⅲ trials of the molecular targeted therapies for ABTC are summarized to provide new insights into clinical practice.Phase II trials have shown that vascular endothelial growth factor receptor inhibitor and mitogen- activated protein kinase inhibitor do not exhibit good anti- tumor activity, but epidermal growth factor receptor (EGFR) inhibitor has proven to be safe and effective in the treatment of ABTC. The only multicenter, open- label, randomized, controlled phase Ⅲ trial has shown that nilotinib combined with gemcitabine and oxaliplatin, as the first- line chemotherapy for ABTC, cannot significantly increase the overall survival in patients. Subgroup analysis has shown that standard chemotherapy combined with nilotinib can significantly increase the progression- free survival in patients. These results indicate that EGFR inhibitor is effective to control the progression of ABTC, suggesting that EGFR might be a novel therapeutic target.