Treatment options for biliary tract cancer: unmet needs, new targets and opportunities from both physicians' and patients' perspectives

Rachna T. Shroff, Melinda Bachini

Research output: Contribution to journalReview articlepeer-review

Abstract

Biliary tract cancer (BTC) is a rare cancer with poor prognosis, characterized by considerable pathophysiological and molecular heterogeneity. While this makes it difficult to treat, it also provides targeted therapy opportunities. Current standard-of-care is chemotherapy ± immunotherapy, but several targeted agents have recently been approved. The current investigational landscape in BTC emphasizes the importance of biomarker testing at diagnosis. MDM2/MDMX are important negative regulators of the tumor suppressor p53 and provide an additional target in BTC (∼5–8% of tumors are MDM2-amplified). Brigimadlin (BI 907828) is a highly potent MDM2-p53 antagonist that has shown antitumor activity in preclinical studies and promising results in early clinical trials; enrollment is ongoing in a potential registrational trial for patients with BTC.

Original languageEnglish (US)
JournalFuture Oncology
DOIs
StateAccepted/In press - 2024

Keywords

  • BI 90782823
  • biliary tract cancer
  • brigimadlin
  • MDM2–p53
  • plain-language summary
  • targeted therapy

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

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