Introduction: Cholangiocarcinoma (CCA) is a devastating liver tumor with a poor prognosis. While less than 50% of the patients with CCA may benefit from surgical resection, the rest undergoes chemotherapy with disappointing results (mean survival <2 years). Alternative pharmacological treatments are needed to improve the outcomes in patients with CCA. Areas covered: In this review, we discuss CCA-related (1) experimental systems used in preclinical studies; (2) pharmacological targets identified by genetic analysis; (3) results obtained in preliminary trials in human with their pros and cons; and (4) possible targeting of endocrinal modulation. A PubMed bibliographic search matching the term ‘cholangiocarcinoma’ with ‘experimental model’, ‘preclinical model’, ‘genetic target’, ‘targeted therapy’, ‘clinical trial’, or ‘translational research’ was conducted and manuscripts published between 2010 and 2020 were retrieved for reading and reviewing. Expert opinion: Several factors contribute to the translational gap between bench research and clinical practice in CCA. The tumor heterogeneity, lack of a preclinical model recapitulating the different features of CCA, and difficult patient enrollment in clinical trials are elements to consider for basic and clinical research in CCA. Establishment of international networks formed by experts in the field of CCA may improve future research and its translational findings on patients.

Cholangiocarcinoma: bridging the translational gap from preclinical to clinical development and implications for future therapy / Baiocchi, L.; Sato, K.; Ekser, B.; Kennedy, L.; Francis, H.; Ceci, L.; Lenci, I.; Alvaro, D.; Franchitto, A.; Onori, P.; Gaudio, E.; Wu, C.; Chakraborty, S.; Glaser, S.; Alpini, G.. - In: EXPERT OPINION ON INVESTIGATIONAL DRUGS. - ISSN 1354-3784. - 30:4(2021), pp. 365-375. [10.1080/13543784.2021.1854725]

Cholangiocarcinoma: bridging the translational gap from preclinical to clinical development and implications for future therapy

Ceci L.;Alvaro D.;Franchitto A.;Onori P.;Gaudio E.;
2021

Abstract

Introduction: Cholangiocarcinoma (CCA) is a devastating liver tumor with a poor prognosis. While less than 50% of the patients with CCA may benefit from surgical resection, the rest undergoes chemotherapy with disappointing results (mean survival <2 years). Alternative pharmacological treatments are needed to improve the outcomes in patients with CCA. Areas covered: In this review, we discuss CCA-related (1) experimental systems used in preclinical studies; (2) pharmacological targets identified by genetic analysis; (3) results obtained in preliminary trials in human with their pros and cons; and (4) possible targeting of endocrinal modulation. A PubMed bibliographic search matching the term ‘cholangiocarcinoma’ with ‘experimental model’, ‘preclinical model’, ‘genetic target’, ‘targeted therapy’, ‘clinical trial’, or ‘translational research’ was conducted and manuscripts published between 2010 and 2020 were retrieved for reading and reviewing. Expert opinion: Several factors contribute to the translational gap between bench research and clinical practice in CCA. The tumor heterogeneity, lack of a preclinical model recapitulating the different features of CCA, and difficult patient enrollment in clinical trials are elements to consider for basic and clinical research in CCA. Establishment of international networks formed by experts in the field of CCA may improve future research and its translational findings on patients.
2021
cholangiocarcinoma; cholangiocyte; endocrine regulation; preclinical model; targeted therapy; translational gap
01 Pubblicazione su rivista::01a Articolo in rivista
Cholangiocarcinoma: bridging the translational gap from preclinical to clinical development and implications for future therapy / Baiocchi, L.; Sato, K.; Ekser, B.; Kennedy, L.; Francis, H.; Ceci, L.; Lenci, I.; Alvaro, D.; Franchitto, A.; Onori, P.; Gaudio, E.; Wu, C.; Chakraborty, S.; Glaser, S.; Alpini, G.. - In: EXPERT OPINION ON INVESTIGATIONAL DRUGS. - ISSN 1354-3784. - 30:4(2021), pp. 365-375. [10.1080/13543784.2021.1854725]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/1469728
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