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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| Format: | Article |
| Language: | English |
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MDPI AG
2022-12-01
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| Series: | International Journal of Molecular Sciences |
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| Online Access: | https://www.mdpi.com/1422-0067/23/23/15124 |
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| author | Maurizio Capuozzo Mariachiara Santorsola Loris Landi Vincenza Granata Francesco Perri Venere Celotto Oreste Gualillo Guglielmo Nasti Alessandro Ottaiano |
| author_facet | Maurizio Capuozzo Mariachiara Santorsola Loris Landi Vincenza Granata Francesco Perri Venere Celotto Oreste Gualillo Guglielmo Nasti Alessandro Ottaiano |
| author_sort | Maurizio Capuozzo |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-09T17:45:08Z |
| format | Article |
| id | doaj.art-0ad8af0688e74ba29e0b9b1720339337 |
| institution | Directory Open Access Journal |
| issn | 1661-6596 1422-0067 |
| language | English |
| last_indexed | 2024-03-09T17:45:08Z |
| publishDate | 2022-12-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Molecular Sciences |
| spelling | doaj.art-0ad8af0688e74ba29e0b9b17203393372023-11-24T11:13:40ZengMDPI AGInternational Journal of Molecular Sciences1661-65961422-00672022-12-0123231512410.3390/ijms232315124Evolution of Treatment in Advanced Cholangiocarcinoma: Old and New towards Precision OncologyMaurizio Capuozzo0Mariachiara Santorsola1Loris Landi2Vincenza Granata3Francesco Perri4Venere Celotto5Oreste Gualillo6Guglielmo Nasti7Alessandro Ottaiano8Coordinamento Farmaceutico, ASL-Naples-3, 80056 Ercolano, ItalyIstituto Nazionale Tumori di Napoli, IRCCS “G. Pascale”, Via M. Semmola, 80131 Naples, ItalySanitary District, Ds. 58 ASL-Naples-3, 80056 Ercolano, ItalyIstituto Nazionale Tumori di Napoli, IRCCS “G. Pascale”, Via M. Semmola, 80131 Naples, ItalyIstituto Nazionale Tumori di Napoli, IRCCS “G. Pascale”, Via M. Semmola, 80131 Naples, ItalyCoordinamento Farmaceutico, ASL-Naples-3, 80056 Ercolano, ItalySERGAS (Servizo Galego de Saude) and IDIS (Instituto de Investigación Sanitaria de Santiago), NEIRID Lab (Neuroendocrine Interactions in Rheumatology and Inflammatory Diseases), Research Laboratory 9, Santiago University Clinical Hospital, 15706 Santiago de Compostela, SpainIstituto Nazionale Tumori di Napoli, IRCCS “G. Pascale”, Via M. Semmola, 80131 Naples, ItalyIstituto Nazionale Tumori di Napoli, IRCCS “G. Pascale”, Via M. Semmola, 80131 Naples, ItalyCholangiocarcinoma (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.https://www.mdpi.com/1422-0067/23/23/15124cholangiocarcinomaivosidenibpemigatinibtarget therapygeneticsprecision oncology |
| spellingShingle | Maurizio Capuozzo Mariachiara Santorsola Loris Landi Vincenza Granata Francesco Perri Venere Celotto Oreste Gualillo Guglielmo Nasti Alessandro Ottaiano Evolution of Treatment in Advanced Cholangiocarcinoma: Old and New towards Precision Oncology International Journal of Molecular Sciences cholangiocarcinoma ivosidenib pemigatinib target therapy genetics precision oncology |
| title | Evolution of Treatment in Advanced Cholangiocarcinoma: Old and New towards Precision Oncology |
| title_full | Evolution of Treatment in Advanced Cholangiocarcinoma: Old and New towards Precision Oncology |
| title_fullStr | Evolution of Treatment in Advanced Cholangiocarcinoma: Old and New towards Precision Oncology |
| title_full_unstemmed | Evolution of Treatment in Advanced Cholangiocarcinoma: Old and New towards Precision Oncology |
| title_short | Evolution of Treatment in Advanced Cholangiocarcinoma: Old and New towards Precision Oncology |
| title_sort | evolution of treatment in advanced cholangiocarcinoma old and new towards precision oncology |
| topic | cholangiocarcinoma ivosidenib pemigatinib target therapy genetics precision oncology |
| url | https://www.mdpi.com/1422-0067/23/23/15124 |
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