Targeted Therapies in Cholangiocarcinoma: Emerging Evidence from Clinical Trials

Cholangiocarcinoma (CCA) is a highly-aggressive malignancy arising from the biliary tree, characterized by a steady increase in incidence globally and a high mortality rate. Most CCAs are diagnosed in the advanced and metastatic phases of the disease, due to the paucity of signs and symptoms in the...

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Main Authors: Maria Maddalena Simile, Paola Bagella, Gianpaolo Vidili, Angela Spanu, Roberto Manetti, Maria Antonietta Seddaiu, Sergio Babudieri, Giordano Madeddu, Pier Andrea Serra, Matteo Altana, Panagiotis Paliogiannis
Format: Article
Language:English
Published: MDPI AG 2019-02-01
Series:Medicina
Subjects:
Online Access:https://www.mdpi.com/1010-660X/55/2/42
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author Maria Maddalena Simile
Paola Bagella
Gianpaolo Vidili
Angela Spanu
Roberto Manetti
Maria Antonietta Seddaiu
Sergio Babudieri
Giordano Madeddu
Pier Andrea Serra
Matteo Altana
Panagiotis Paliogiannis
author_facet Maria Maddalena Simile
Paola Bagella
Gianpaolo Vidili
Angela Spanu
Roberto Manetti
Maria Antonietta Seddaiu
Sergio Babudieri
Giordano Madeddu
Pier Andrea Serra
Matteo Altana
Panagiotis Paliogiannis
author_sort Maria Maddalena Simile
collection DOAJ
description Cholangiocarcinoma (CCA) is a highly-aggressive malignancy arising from the biliary tree, characterized by a steady increase in incidence globally and a high mortality rate. Most CCAs are diagnosed in the advanced and metastatic phases of the disease, due to the paucity of signs and symptoms in the early stages. This fact, along with the poor results of the local and systemic therapies currently employed, is responsible for the poor outcome of CCA patients and strongly supports the need for novel therapeutic agents and strategies. In recent years, the introduction of next-generation sequencing technologies has opened new horizons for a better understanding of the genetic pathophysiology of CCA and, consequently, for the identification and evaluation of new treatments tailored to the molecular features or alterations progressively elucidated. In this review article, we describe the potential targets under investigation and the current molecular therapies employed in biliary tract cancers. In addition, we summarize the main drugs against CCA under evaluation in ongoing trials and describe the preliminary data coming from these pioneering studies.
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spelling doaj.art-a834510c0d2248399865e98a8605f0212023-09-03T04:54:57ZengMDPI AGMedicina1010-660X2019-02-015524210.3390/medicina55020042medicina55020042Targeted Therapies in Cholangiocarcinoma: Emerging Evidence from Clinical TrialsMaria Maddalena Simile0Paola Bagella1Gianpaolo Vidili2Angela Spanu3Roberto Manetti4Maria Antonietta Seddaiu5Sergio Babudieri6Giordano Madeddu7Pier Andrea Serra8Matteo Altana9Panagiotis Paliogiannis10Department of Medical, Surgical, and Experimental Sciences, University of Sassari, Viale San Pietro 43, 07100 Sassari, ItalyDepartment of Medical, Surgical, and Experimental Sciences, University of Sassari, Viale San Pietro 43, 07100 Sassari, ItalyDepartment of Medical, Surgical, and Experimental Sciences, University of Sassari, Viale San Pietro 43, 07100 Sassari, ItalyDepartment of Medical, Surgical, and Experimental Sciences, University of Sassari, Viale San Pietro 43, 07100 Sassari, ItalyDepartment of Medical, Surgical, and Experimental Sciences, University of Sassari, Viale San Pietro 43, 07100 Sassari, ItalyDepartment of Medical, Surgical, and Experimental Sciences, University of Sassari, Viale San Pietro 43, 07100 Sassari, ItalyDepartment of Medical, Surgical, and Experimental Sciences, University of Sassari, Viale San Pietro 43, 07100 Sassari, ItalyDepartment of Medical, Surgical, and Experimental Sciences, University of Sassari, Viale San Pietro 43, 07100 Sassari, ItalyDepartment of Medical, Surgical, and Experimental Sciences, University of Sassari, Viale San Pietro 43, 07100 Sassari, ItalyDepartment of Medical, Surgical, and Experimental Sciences, University of Sassari, Viale San Pietro 43, 07100 Sassari, ItalyDepartment of Medical, Surgical, and Experimental Sciences, University of Sassari, Viale San Pietro 43, 07100 Sassari, ItalyCholangiocarcinoma (CCA) is a highly-aggressive malignancy arising from the biliary tree, characterized by a steady increase in incidence globally and a high mortality rate. Most CCAs are diagnosed in the advanced and metastatic phases of the disease, due to the paucity of signs and symptoms in the early stages. This fact, along with the poor results of the local and systemic therapies currently employed, is responsible for the poor outcome of CCA patients and strongly supports the need for novel therapeutic agents and strategies. In recent years, the introduction of next-generation sequencing technologies has opened new horizons for a better understanding of the genetic pathophysiology of CCA and, consequently, for the identification and evaluation of new treatments tailored to the molecular features or alterations progressively elucidated. In this review article, we describe the potential targets under investigation and the current molecular therapies employed in biliary tract cancers. In addition, we summarize the main drugs against CCA under evaluation in ongoing trials and describe the preliminary data coming from these pioneering studies.https://www.mdpi.com/1010-660X/55/2/42cancerbiliary treecholangiocarcinomamolecular pathwaysgenomicstargeted therapies
spellingShingle Maria Maddalena Simile
Paola Bagella
Gianpaolo Vidili
Angela Spanu
Roberto Manetti
Maria Antonietta Seddaiu
Sergio Babudieri
Giordano Madeddu
Pier Andrea Serra
Matteo Altana
Panagiotis Paliogiannis
Targeted Therapies in Cholangiocarcinoma: Emerging Evidence from Clinical Trials
Medicina
cancer
biliary tree
cholangiocarcinoma
molecular pathways
genomics
targeted therapies
title Targeted Therapies in Cholangiocarcinoma: Emerging Evidence from Clinical Trials
title_full Targeted Therapies in Cholangiocarcinoma: Emerging Evidence from Clinical Trials
title_fullStr Targeted Therapies in Cholangiocarcinoma: Emerging Evidence from Clinical Trials
title_full_unstemmed Targeted Therapies in Cholangiocarcinoma: Emerging Evidence from Clinical Trials
title_short Targeted Therapies in Cholangiocarcinoma: Emerging Evidence from Clinical Trials
title_sort targeted therapies in cholangiocarcinoma emerging evidence from clinical trials
topic cancer
biliary tree
cholangiocarcinoma
molecular pathways
genomics
targeted therapies
url https://www.mdpi.com/1010-660X/55/2/42
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