Contents
Special Issue Topic

Precision Medicine for Cholangiocarcinoma

Guest Editor

Prof. Lorenza Rimassa E-Mail

Associate Professor of Medical Oncology, Department of Biomedical Sciences, Humanitas University; Deputy Director, Medical Oncology Unit, Humanitas Cancer Center, Humanitas Research Hospital, IRCCS, Milano, Italy

Research Keywords: liver cancer; hepatocellular carcinoma (HCC); cholangiocarcinoma (CCA)/biliary tract cancer (BTC); biomarkers; clinical trials

About the Special lssue

The incidence of cholangiocarcinoma (CCA) is increasing in the last years, patients are frequently diagnosed with advanced disease, relapse rates are high also in those undergoing surgery, thus prognosis remains poor. Unfortunately, treatment options have been historically limited, and chemotherapy is still the standard of care in both the adjuvant and advanced setting. However, more recently, different subtypes of CCA have been defined based on the anatomical site and genetic and/or epigenetic aberrations. Thanks to the improving knowledge of CCA biology and molecular heterogeneity, especially for intrahepatic CCA, novel therapeutic targets have been identified and molecularly targeted agents have shown evidence of activity with a good safety profile. Fibroblast growth factor receptor 2 gene fusions and isocitrate dehydrogenase 1 and 2 mutations are the most studied targets and novel agents have been demonstrated active in phase 2 and 3 trials in patients with CCA harboring these aberrations. In addition, other pathways are being tested not only in intrahepatic CCA but also in other subtypes of biliary tract cancers. Finally, immunological characterization of the tumor and the microenvironment, albeit with mixed results, is paving the way for the development of potential immunotherapeutic strategies. In summary, the growing knowledge of CCA biology will allow to develop new therapeutic approaches that will deeply change the treatment paradigm for this disease in the near future.
This special issue is focused on precision medicine for patients with CCA, including new targets, emerging therapies, resistance mechanisms, open issues, and future perspectives, with the aim to improve the management and the prognosis of patients with CCA.

Keywords: cholangiocarcinoma; biliary tract cancer; molecular heterogeneity; genetic/epigenetic aberrations; novel targets; immune microenvironment; novel agents

Published Articles

Open Access Case Report
Cystic presentation of primary hepatic neuroendocrine tumour: a case report with a brief review of literature
Mangesh Londhe ... Charusheela Gore
Published: April 26, 2023 Explor Target Antitumor Ther. 2023;4:266–272
1528 16 1
Open Access Review
Genomic alterations in cholangiocarcinoma: clinical significance and relevance to therapy
Marianeve Carotenuto ... Nicola Normanno
Published: April 26, 2022 Explor Target Antitumor Ther. 2022;3:200–223
5303 85 15
Open Access Case Report
Liver venous deprivation for resection of advanced hilar cholangiocarcinoma—a case report and review of the literature
Radoslava Stoyanova ... Alexander Klaus
Published: February 28, 2022 Explor Target Antitumor Ther. 2022;3:90–96
2292 52 1
Open Access Review
A review of molecularly targeted therapy in biliary tract carcinoma: what is the next step?
Giacomo Aimar ... Massimo Di Maio
Published: October 31, 2021 Explor Target Antitumor Ther. 2021;2:448–464
3395 83 4
Open Access Review
Current options and future directions of systemic therapy for advanced biliary tract cancer
Maria Giuseppina Prete ... Lorenza Rimassa
Published: October 31, 2021 Explor Target Antitumor Ther. 2021;2:416–433
4034 81 1
Open Access Review
Targeting protein kinase CK2 in the treatment of cholangiocarcinoma
Padma-Sheela Jayaraman, Kevin Gaston
Published: October 31, 2021 Explor Target Antitumor Ther. 2021;2:434–447
3318 69 2