Evolution of Treatment in Advanced Cholangiocarcinoma: Old and New towards Precision Oncology

Cholangiocarcinoma (CCA) is a malignant neoplasm arising in the epithelium of the biliary tract. It represents the second most common primary liver cancer in the world, after hepatocellular carcinoma, and it constitutes 10–15% of hepatobiliary neoplasms and 3% of all gastrointestinal tumors. As in o...

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Bibliographic Details
Main Authors: Maurizio Capuozzo, Mariachiara Santorsola, Loris Landi, Vincenza Granata, Francesco Perri, Venere Celotto, Oreste Gualillo, Guglielmo Nasti, Alessandro Ottaiano
Format: Article
Language:English
Published: MDPI AG 2022-12-01
Series:International Journal of Molecular Sciences
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Online Access:https://www.mdpi.com/1422-0067/23/23/15124
Description
Summary:Cholangiocarcinoma (CCA) is a malignant neoplasm arising in the epithelium of the biliary tract. It represents the second most common primary liver cancer in the world, after hepatocellular carcinoma, and it constitutes 10–15% of hepatobiliary neoplasms and 3% of all gastrointestinal tumors. As in other types of cancers, recent studies have revealed genetic alterations underlying the establishment and progression of CCA. The most frequently involved genes are <i>APC</i>, <i>ARID1A</i>, <i>AXIN1</i>, <i>BAP1</i>, <i>EGFR</i>, <i>FGFRs</i>, <i>IDH1/2</i>, <i>RAS</i>, <i>SMAD4</i>, and <i>TP53</i>. Actionable targets include alterations of FGFRs, IDH1/2, BRAF, NTRK, and HER2. “Precision oncology” is emerging as a promising approach for CCA, and it is possible to inhibit the altered function of these genes with molecularly oriented drugs (pemigatinib, ivosidenib, vemurafenib, larotrectinib, and trastuzumab). In this review, we provide an overview of new biologic drugs (their structures, mechanisms of action, and toxicities) to treat metastatic CCA, providing readers with panoramic information on the trajectory from “old” chemotherapies to “new” target-oriented drugs.
ISSN:1661-6596
1422-0067