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Review
. 2019 Feb 8;55(2):42.
doi: 10.3390/medicina55020042.

Targeted Therapies in Cholangiocarcinoma: Emerging Evidence from Clinical Trials

Maria Maddalena Simile  1 , Paola Bagella, Gianpaolo Vidili, Angela Spanu, Roberto Manetti, Maria Antonietta Seddaiu, Sergio Babudieri, Giordano Madeddu, Pier Andrea Serra, Matteo Altana, Panagiotis Paliogiannis
Affiliations
Review

Targeted Therapies in Cholangiocarcinoma: Emerging Evidence from Clinical Trials

Maria Maddalena Simile et al. Medicina (Kaunas). .

Abstract

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.

Keywords: biliary tree; cancer; cholangiocarcinoma; genomics; molecular pathways; targeted therapies.

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Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure 1
Figure 1
Chemical structures of the main tyrosine kinase inhibitors currently under investigation for targeted treatment of cholangiocarcinoma.
Figure 2
Figure 2
Chemical structures of promising agents included in ongoing clinical trials for targeted treatment of cholangiocarcinoma.
Figure 3
Figure 3
Overview of some of the molecular pathways deregulated in cholangiocarcinoma and the available inhibitors of these cascades.
See this image and copyright information in PMC

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