Division of Hematology Oncology, Department of Medicine, Mayo Clinic, Scottsdale, AZ 85259, USA.
Department of Cancer Biology, Mayo Clinic, Jacksonville, FL 32224, USA.
Division of Gastroenterology & Hepatology, Department of Medicine, Mayo Clinic, Scottsdale, AZ 85259, USA.
Department of Molecular Medicine, Department of Laboratory Medicine & Pathology, Mayo Clinic, Rochester, MN 55902, USA.
Division of Hepatobiliary & Pancreatic Surgery, Department of Surgery, Mayo Clinic, Rochester, MN 55902, USA.
Division of Interventional Radiology, Department of Radiology, Mayo Clinic, Scottsdale, AZ 85259, USA.
Worldwide hepatobiliary cancers are the second leading cause of cancer related death. Despite their relevance, hepatobiliary cancers have a paucity of approved systemic therapy options. However, there are a number of emerging therapeutic biomarkers and therapeutic concepts that show promise. In hepatocellular carcinoma, nivolumab appears particularly promising and recently received US FDA approval. In intrahepatic cholangiocarcinoma, therapies targeting FGFR2 and IDH1 and immune checkpoint inhibitors are the furthest along and generating the most excitement. There are additional biomarkers that merit further exploration in hepatobiliary cancers including FGF19, ERRFI1, TERT, BAP1, BRAF, CDKN2A, tumor mutational burden and ERBB2 (HER2/neu). Development of new and innovative therapies would help address the unmet need for effective systemic therapies in advanced and metastatic hepatobiliary cancers.