Targeting mTOR signaling for the treatment of intrahepatic cholangiocarcinoma with TSC1/ARID1A mutations: a case report with an unexpected response.

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Date publication

septembre 2024

Journal

Therapeutic advances in medical oncology

Auteurs

Membres identifiés du Cancéropôle Est :
Dr BOIDOT Romain, Pr BORG Christophe, Pr GHIRINGHELLI François, Dr VIENOT Angélique


Tous les auteurs :
Daugan C, Boidot R, Ghiringhelli F, Borg C, Vienot A

Résumé

Biliary tract cancer incidence is increasing and the prognostic remains dismal. The development of personalized medicine is a pivotal issue in proposing therapeutic options for biliary tract cancer patients. Whole exome sequencing identifies approximately 15% of mutations and 15% of fusions in intrahepatic cholangiocarcinoma. Other patients are not currently eligible for targeted therapy. Here, we present a patient treated for a metastatic cholangiocarcinoma with an unexpected response to a mammalian target of rapamycin (mTOR) targeting agent. Whole exome sequencing enabled the identification of and mutations. Reintroduction of mTOR inhibitors with similar results sustains the main role of these targeted agents in the control of the disease. These results suggest the existence of an mTOR oncogenic addiction in biliary tract cancer. Our results support the interest in performing exome sequencing in liver cancers and the potential to identify actionable mutations with important therapeutic issues.

Mots clés

ARID1A, TSC1, case report, cholangiocarcinoma, mTOR inhibitors

Référence

Ther Adv Med Oncol. 2024 09 13;16:17588359241271793