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

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.

SUBMITTER: Shroff RT

PROVIDER: S-EPMC11376410 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature

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

Shroff Rachna T RT Bachini Melinda M

Future oncology (London, England) 20240530 20

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 ...[more]

PMID: 38861288

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Dataset Information

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

Publications

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

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