A perspective on molecular therapy in cholangiocarcinoma: present status and future directions.
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.
National Cancer Institute, Laboratory of Experimental Carcinogenesis, NIH, Building 37, Room 4146A, 37 Convent Drive, Bethesda, MD 20892-4262, USA ; Biotech Research & Innovation Centre, University of Copenhagen, DK-2200 Copenhagen, Denmark.
Jesper B
JB
Andersen
National Cancer Institute, Laboratory of Experimental Carcinogenesis, NIH, Building 37, Room 4146A, 37 Convent Drive, Bethesda, MD 20892-4262, USA.
Snorri S
SS
Thorgeirsson