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Review
. 2024;20(20):1435-1450.
doi: 10.1080/14796694.2024.2340959. Epub 2024 May 30.

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

Rachna T Shroff  1 , Melinda Bachini  2
Affiliations
Review

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

Rachna T Shroff et al. Future Oncol. 2024.

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.

Keywords: BI 90782823; MDM2–p53; biliary tract cancer; brigimadlin; plain-language summary; targeted therapy.

Plain language summary

[Box: see text].

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Conflict of interest statement

The authors have no competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Figures

Infographic:
Infographic:
A PDF version of this infographic is available as supplemental material.
Figure 1.
Figure 1.
Framework for the approach to managing biliary tract cancer. 2L: Second-line treatment; dMMR: Deficient mismatch repair; MSI-H: Microsatellite instability-high; NGS: Next-generation sequencing; PS: Performance score. Adapted from Scott et al. 2022 with permission from [3].
Figure 2.
Figure 2.
Waterfall plot of BTC patients included in the ongoing brigimadlin monotherapy (NCT03449381) or combination therapy (NCT03964233) trials. Reused from Yamamoto N, et al. 2024 [98] Licensed under Attribution-NonCommercial-NoDerivatives 4.0 Unported License (https://creativecommons.org/licenses/by-nc-nd/4.0/), with no modifications. BTC: Biliary tract cancer; CN: Copy number; PR: Partial response; UN: Unknown.
Figure 3.
Figure 3.
Study design of BRIGHTLINE-2. Reuse from Yoo C et al. 2024 [100]. Licensed under Attribution-NonCommercial-NoDerivatives 4.0 Unported License (https://creativecommons.org/licenses/by-nc-nd/4.0/), with no modifications.
See this image and copyright information in PMC

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