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Marine Valéry¹, Baptiste Cervantes¹, Cristina Smolenschi²

¹Gustave Roussy, Département de médecine oncologique, F-94805, VILLEJUIF France.

Bulletin Du Cancer

|December 19, 2022

Summary

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Targeting DNA mutations in biliary tract cancers (BTC) offers new hope. This review explores actionable targets like IDH1 mutations and KRASG12C, aiming to improve prognosis for these rare, aggressive cancers.

Area of Science:

Oncology
Genetics
Molecular Biology

Context:

Biliary tract cancers (BTC) have a poor prognosis, with limited treatment options and low survival rates.
Current chemotherapy offers modest survival benefits, highlighting the need for novel therapeutic strategies.
Intrahepatic cholangiocarcinoma exhibits a high rate of somatic alterations, presenting opportunities for targeted therapies.

Purpose:

To review targetable DNA mutations in BTC.
To discuss emerging molecular alterations and their corresponding targeted therapies.
To explore new therapeutic perspectives for improving BTC patient outcomes.

Summary:

BTC harbors numerous targetable genetic alterations, including IDH1 mutations and FGFR2 fusions.
Approved therapies like ivosidenib and pemigatinib target specific mutations, but other alterations (e.g., BRAF, HER2, KRASG12C) are also being investigated.

Keywords:

Altération moléculaire Biliary tract cancer Cancer des voies Cholangiocarcinoma Cholangiocarcinome Mutation Targetable alteration biliaires ciblable

Related Experiment Videos

This review focuses on actionable mutations in BTC and the molecules being developed to treat them.

Impact:

Identifies key molecular targets for BTC treatment.
Highlights the potential of precision medicine to improve survival in BTC.
Offers a comprehensive overview of current and future targeted therapies for biliary tract cancers.