Drug therapy for biliary tract cancer has long been developed mainly with cytotoxic agents, and its effectiveness has been evaluated each biliary site together. However, the differences in clinical conditions and genetic alterations for each biliary sites have been clarified. Biliary tract cancer is also characterized by many genetic alterations that can be target for molecular-targeted agents. Therefore, there are high expectations for precision medicine based on genetic alterations for the treatment of biliary tract cancer. Several effectiveness of molecular-targeted agents and immunotherapy based on genetic alterations have been reported so far. In recent years, gene panel tests have become widespread in Japan, and precision medicine based on gene alterations identified from comprehensive genomic profiling has become possible in daily clinical practice. On the other hand, there are many issues to be solved, such as the system for providing matched therapeutic drugs for the patients, the difficulty of collecting enough tissues for comprehensive genomic profiling, and the problem of acquisition resistance due to the use of molecular-target drugs. Therefore, further development is needed to solve these problems in the future.
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