A perspective on molecular therapy in cholangiocarcinoma: present status and future directions

Jesper Bøje Andersen; Snorri S Thorgeirsson

doi:10.2217/hep.13.4

A perspective on molecular therapy in cholangiocarcinoma: present status and future directions

Jesper Bøje Andersen, Snorri S Thorgeirsson

Abstract

Cholangiocarcinoma (CCA) is an orphan cancer with limited understanding of its genetic and genomic pathogenesis. Typically, it is highly treatment-refractory and patient outcome is dismal. Currently, there are no approved therapeutics for CCA and surgical resection remains the only option with curative intent. Clinical trials are currently being performed in a mixed cohort of biliary tract cancers that includes intrahepatic CCA, extrahepatic/perihilar CCA, distal extrahepatic CCA, gallbladder carcinoma and, in rare cases, even pancreatic cancers. Today, clinical trials fail primarily because they are underpowered mixed cohorts and designed without intent to enrich for markers to optimize success for targeted therapy. This review aims to emphasize current clinical attempts for targeted therapy of CCA, as well as highlight promising new candidate pathways revealed by translational genomics.

Originalsprog	Engelsk
Tidsskrift	Hepatic Oncology
Vol/bind	1
Udgave nummer	1
Sider (fra-til)	143-157
Antal sider	15
ISSN	2045-0923
DOI	https://doi.org/10.2217/hep.13.4
Status	Udgivet - 2014

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A perspective on molecular therapy in cholangiocarcinoma: present status and future directionsForlagets udgivne version, 7 MB

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title = "A perspective on molecular therapy in cholangiocarcinoma: present status and future directions",

abstract = "Cholangiocarcinoma (CCA) is an orphan cancer with limited understanding of its genetic and genomic pathogenesis. Typically, it is highly treatment-refractory and patient outcome is dismal. Currently, there are no approved therapeutics for CCA and surgical resection remains the only option with curative intent. Clinical trials are currently being performed in a mixed cohort of biliary tract cancers that includes intrahepatic CCA, extrahepatic/perihilar CCA, distal extrahepatic CCA, gallbladder carcinoma and, in rare cases, even pancreatic cancers. Today, clinical trials fail primarily because they are underpowered mixed cohorts and designed without intent to enrich for markers to optimize success for targeted therapy. This review aims to emphasize current clinical attempts for targeted therapy of CCA, as well as highlight promising new candidate pathways revealed by translational genomics.",

author = "Andersen, {Jesper B{\o}je} and Thorgeirsson, {Snorri S}",

year = "2014",

doi = "10.2217/hep.13.4",

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T2 - present status and future directions

AU - Andersen, Jesper Bøje

AU - Thorgeirsson, Snorri S

PY - 2014

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N2 - Cholangiocarcinoma (CCA) is an orphan cancer with limited understanding of its genetic and genomic pathogenesis. Typically, it is highly treatment-refractory and patient outcome is dismal. Currently, there are no approved therapeutics for CCA and surgical resection remains the only option with curative intent. Clinical trials are currently being performed in a mixed cohort of biliary tract cancers that includes intrahepatic CCA, extrahepatic/perihilar CCA, distal extrahepatic CCA, gallbladder carcinoma and, in rare cases, even pancreatic cancers. Today, clinical trials fail primarily because they are underpowered mixed cohorts and designed without intent to enrich for markers to optimize success for targeted therapy. This review aims to emphasize current clinical attempts for targeted therapy of CCA, as well as highlight promising new candidate pathways revealed by translational genomics.

AB - Cholangiocarcinoma (CCA) is an orphan cancer with limited understanding of its genetic and genomic pathogenesis. Typically, it is highly treatment-refractory and patient outcome is dismal. Currently, there are no approved therapeutics for CCA and surgical resection remains the only option with curative intent. Clinical trials are currently being performed in a mixed cohort of biliary tract cancers that includes intrahepatic CCA, extrahepatic/perihilar CCA, distal extrahepatic CCA, gallbladder carcinoma and, in rare cases, even pancreatic cancers. Today, clinical trials fail primarily because they are underpowered mixed cohorts and designed without intent to enrich for markers to optimize success for targeted therapy. This review aims to emphasize current clinical attempts for targeted therapy of CCA, as well as highlight promising new candidate pathways revealed by translational genomics.

U2 - 10.2217/hep.13.4

DO - 10.2217/hep.13.4

M3 - Review

C2 - 24772266

SN - 2045-0923

VL - 1

SP - 143

EP - 157

JO - Hepatic Oncology

JF - Hepatic Oncology

IS - 1

ER -

A perspective on molecular therapy in cholangiocarcinoma: present status and future directions

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