Emerging molecular target antagonists for the treatment of biliary tract cancer

Pasquale Lombardi, Donatella Marino, Elisabetta Fenocchio, Giovanna Chilà, Massimo Aglietta, Francesco Leone

Research output: Contribution to journalReview articlepeer-review


Introduction: Biliary tract cancers (BTCs) are a heterogeneous group of cancers, characterized by low incidence but poor prognosis. Even after complete surgical resection for early stage, relapse is frequent and the lack of effective treatments contributes to the dismal prognosis. To date, the only standard treatment in first-line is cisplatin/gemcitabine combination, whereas no standard in 2nd-line has been defined. Hence, the current goal is to better understand the biology of BTCs, discovering new treatment methods and improving clinical outcomes. Areas covered: The development of next-generation-sequencing has unveiled the picture of the molecular signatures characterizing BTCs, leading to the identification of actionable mutations in biomarker-driven clinical trials. In this review we will cover the genetic landscape of BTC, focusing on the efficacy of existing treatments. Furthermore, we will discuss emerging molecular targets and evaluate the findings of pre-clinical studies. Finally, the encouraging results of clinical trials involving targeted therapies or immunotherapy will be reviewed. Expert opinion: FGFR fusion rearrangements and IDH1 or IDH2 mutations are the most promising targeted treatments under evaluation. In addition, innovative trial design will allow to offer a chance for tailored medicine to infrequent subgroups of BTCs patients based on their molecular features rather than their histology.

Original languageEnglish
Pages (from-to)63-75
Number of pages13
JournalExpert Opinion on Emerging Drugs
Issue number1
Publication statusPublished - Jan 2 2018


  • Biliary tract cancer
  • cholangiocarcinoma
  • emerging drugs
  • FGFR
  • growth factor receptor
  • IDH1
  • immunotherapy
  • molecular targeted drugs

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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