Emerging treatment with systemic chemotherapy and targeted agents for biliary cancers.

Clinically, 5-fluorouracil (5-FU) and its derivatives, platinum-based agents and gemcitabine (GEM) are used as key treatments for unresectable or recurrent biliary cancer. A phase III clinical trial has demonstrated that treatment with GEM plus CDDP prolongs the survival of patients with biliary cancer compared with treatment with GEM alone. Moreover, promising results are also being reported against molecular targets, particularly with inhibitors of EGFR. In addition, the development of novel drugs based on biological markers might be important for the treatment of biliary cancer. In the future, a paradigm shift from disease-specific drug development to biomarker-oriented investigations may be applicable for rare diseases such as biliary cancer.