Novel targeted therapy strategies for biliary tract cancers and hepatocellular carcinoma

Thomas T. Deleon, Daniel Ahn, James M. Bogenberger, Panagiotis Z Anastasiadis, Mansi Arora, Ramesk K Ramanathan, Bashar A. Aqel, George Vasmatzis, Mark Truty, Rahmi Oklu, Tanios Bekaii-Saab, Mitesh J Borad

Research output: Contribution to journalReview articlepeer-review

16 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)553-566
Number of pages14
JournalFuture Oncology
Issue number6
StatePublished - Mar 1 2018


  • biomarkers
  • cholangiocarcinoma
  • gallbladder cancer
  • hepatocellular cancer
  • immunotherapy
  • targeted therapy

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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