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Advances in Human Induced Pluripotent Stem Cell-Derived Chimeric Antigen Receptor-Expressing Natural Killer Cells

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Evolving precision: Updates in targeted therapy for cholangiocarcinoma.

Giulia Tesini^1,2, Halima Ibrahim³, Lorenza Rimassa^2,4

¹School of Cancer Sciences, University of Glasgow, Glasgow, UK.

Hepatology (Baltimore, Md.)

|September 22, 2025

Summary

Next-Generation Sequencing (NGS) identifies actionable targets in biliary tract cancer (BTC). Targeted drugs offer clinical benefit and are recommended for advanced BTC patients post-first-line treatment.

Keywords:

biliary tract cancer drug resistance molecular diagnostic techniques molecular targeted therapy new actionable targets

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Area of Science:

Oncology
Genomics
Precision Medicine

Background:

Biliary tract cancer (BTC) treatment has advanced with genomic profiling.
Next-Generation Sequencing (NGS) identifies actionable molecular alterations in ~50% of BTC patients.
Intrahepatic cholangiocarcinoma shows the highest incidence of these alterations.

Purpose of the Study:

To review the clinical utility of targeted drugs in advanced BTC.
To compare targeted drug trial results with clinical practice.
To outline single-gene testing strategies for NGS-inaccessible cases.

Main Methods:

Review of clinical trial data for targeted therapies in BTC.
Analysis of single-gene testing yields and therapeutic implications.
Discussion of future directions in precision medicine for BTC.

Main Results:

Targeted drugs provide clinical benefit with manageable safety profiles.
Targeted therapies are recommended for advanced BTC patients after first-line treatment failure.
NGS is crucial for identifying actionable alterations, guiding treatment decisions.

Conclusions:

Precision medicine, particularly targeted therapies, is transforming BTC treatment.
Integrating targeted drugs into treatment algorithms requires careful consideration of resistance mechanisms and new targets.
Future research should focus on novel strategies and newly identified actionable alterations for improved patient outcomes.