Cholangiocarcinoma: Molecular pathways and therapeutic opportunities

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73 Scopus citations

Abstract

Cholangiocarcinoma (CCA) is an aggressive biliary tract malignancy with limited treatment options and low survival rates. Currently, there are no curative medical therapies for CCA. Recent advances have enhanced our understanding of the genetic basis of this disease, and elucidated therapeutically relevant targets. Therapeutic efforts in development are directed at several key pathways due to genetic aberrations including receptor tyrosine kinase pathways, mutant IDH enzymes, the PI3K-AKT-mTOR pathway, and chromatin remodeling networks. A highly desmoplastic, hypovascular stroma is characteristic of CCAs and recent work has highlighted the importance of targeting this pathway via stromal myofibroblast depletion. Future efforts should concentrate on combination therapies with action against the cancer cell and the surrounding tumor stroma. As the mutational landscape of CCA is being illuminated, molecular profiling of patient tumors will enable identification of specific mutations and the opportunity to offer directed, personalized treatment options.

Original languageEnglish (US)
Pages (from-to)456-464
Number of pages9
JournalSeminars in liver disease
Volume34
Issue number4
DOIs
StatePublished - Nov 1 2014

Keywords

  • cholangiocarcinoma
  • molecular pathogenesis
  • targeted therapy

ASJC Scopus subject areas

  • Hepatology

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