Criteria for preclinical models of cholangiocarcinoma: scientific and medical relevance

CCA Model Consortium

doi:10.1038/s41575-022-00739-y

Criteria for preclinical models of cholangiocarcinoma: scientific and medical relevance

CCA Model Consortium

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15 Scopus citations

Abstract

Cholangiocarcinoma (CCA) is a rare malignancy that develops at any point along the biliary tree. CCA has a poor prognosis, its clinical management remains challenging, and effective treatments are lacking. Therefore, preclinical research is of pivotal importance and necessary to acquire a deeper understanding of CCA and improve therapeutic outcomes. Preclinical research involves developing and managing complementary experimental models, from in vitro assays using primary cells or cell lines cultured in 2D or 3D to in vivo models with engrafted material, chemically induced CCA or genetically engineered models. All are valuable tools with well-defined advantages and limitations. The choice of a preclinical model is guided by the question(s) to be addressed; ideally, results should be recapitulated in independent approaches. In this Consensus Statement, a task force of 45 experts in CCA molecular and cellular biology and clinicians, including pathologists, from ten countries provides recommendations on the minimal criteria for preclinical models to provide a uniform approach. These recommendations are based on two rounds of questionnaires completed by 35 (first round) and 45 (second round) experts to reach a consensus with 13 statements. An agreement was defined when at least 90% of the participants voting anonymously agreed with a statement. The ultimate goal was to transfer basic laboratory research to the clinics through increased disease understanding and to develop clinical biomarkers and innovative therapies for patients with CCA.

Original language	English (US)
Pages (from-to)	462-480
Number of pages	19
Journal	Nature Reviews Gastroenterology and Hepatology
Volume	20
Issue number	7
DOIs	https://doi.org/10.1038/s41575-022-00739-y
State	Published - Jul 2023
Externally published	Yes

Bibliographical note

Funding Information:
The authors thank the European Network for the Study of Cholangiocarcinoma (ENS-CCA) and the European H2020 COST Action CA18122. The authors also acknowledge the valuable contributions of the external advisory panel. C.C. is supported by Inserm, Université de Rennes 1 and by a grant from the French Ministry of Health and the French National Cancer Institute (PRT-K20-136), CHU Rennes, CLCC Eugène Marquis, Rennes. M.M.A.V. and L.J.W.v.d.L. are supported by Medical Delta Regenerative Medicine 4D (Generating complex tissues with stem cells and printing technology) and TKI-LSH grant EMC-LSH19002. S.R. is supported by the German Research Foundation (DFG, project no. 314905040 and no. 493697503) and by German Cancer Aid (Deutsche Krebshilfe, project no. 70113922). S.V. is supported by Ministerio de Ciencia, Innovación y Universidades, Convocatoria 2019 para incentivar la Incorporación Estable de Doctores (IED2019-001007-I), by FEDER/Ministerio de Ciencia, Innovación y Universidades – Agencia Estatal de Investigación (PID2020‐116344‐RB‐100) and by the Government of Navarra-Health Research Department (58; 2018). J.V. is funded by Ministerio de Ciencia e Innovación, part of Agencia Estatal de Investigación (AEI), through the Retos Investigación grant number PID2019-108651RJ-I00/DOI 10.13039/501100011033. The authors thank CERCA Programme/Generalitat de Catalunya for institutional support. R.I.R.M. and J.J.G.M. are supported by Instituto de Salud Carlos III, Spain (PI20/00189, PI19/00819) co-funded by the European Union. L. Fouassier belongs to a team supported by the Fondation pour la Recherche Médicale (Equipe FRM 2020 no. EQU202003010517) and is supported by Inserm and Sorbonne Université, INCa and ITMO Cancer of Aviesan within the framework of the 2021–2030 Cancer Control Strategy, on funds administered by Inserm.

Funding Information:
C.B. receives honoraria from Incyte, Servier, BI and AstraZeneca, and research funding from Avacta and Medannex. A.F. received consultancy fees from Bayer, AstraZeneca, Roche, Boston, Exact Science and Guerbert. J.W.V. reports honoraria from Agios, AstraZeneca, Genoscience Pharma, Incyte, Mundipharma EDO, Mylan, QED, Servier, SIRTex and Zymeworks, and honoraria and non-financial support from NuCana, all outside the submitted work. The other authors declare no competing interests.

Publisher Copyright:
© 2023, Springer Nature Limited.

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Journal Article
Review
Research Support, Non-U.S. Gov't

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10.1038/s41575-022-00739-y

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@article{22ec9479cdec401cb146ae1e0681273d,

title = "Criteria for preclinical models of cholangiocarcinoma: scientific and medical relevance",

abstract = "Cholangiocarcinoma (CCA) is a rare malignancy that develops at any point along the biliary tree. CCA has a poor prognosis, its clinical management remains challenging, and effective treatments are lacking. Therefore, preclinical research is of pivotal importance and necessary to acquire a deeper understanding of CCA and improve therapeutic outcomes. Preclinical research involves developing and managing complementary experimental models, from in vitro assays using primary cells or cell lines cultured in 2D or 3D to in vivo models with engrafted material, chemically induced CCA or genetically engineered models. All are valuable tools with well-defined advantages and limitations. The choice of a preclinical model is guided by the question(s) to be addressed; ideally, results should be recapitulated in independent approaches. In this Consensus Statement, a task force of 45 experts in CCA molecular and cellular biology and clinicians, including pathologists, from ten countries provides recommendations on the minimal criteria for preclinical models to provide a uniform approach. These recommendations are based on two rounds of questionnaires completed by 35 (first round) and 45 (second round) experts to reach a consensus with 13 statements. An agreement was defined when at least 90% of the participants voting anonymously agreed with a statement. The ultimate goal was to transfer basic laboratory research to the clinics through increased disease understanding and to develop clinical biomarkers and innovative therapies for patients with CCA.",

author = "{CCA Model Consortium} and Calvisi, {Diego F.} and Luke Boulter and Javier Vaquero and Anna Saborowski and Luca Fabris and Rodrigues, {Pedro M.} and C{\'e}dric Coulouarn and Castro, {Rui E.} and Oreste Segatto and Chiara Raggi and {van der Laan}, {Luc J.W.} and Guido Carpino and Benjamin Goeppert and Stephanie Roessler and Kendall, {Timothy J.} and Matthias Evert and Ester Gonzalez-Sanchez and Valle, {Juan W.} and Arndt Vogel and John Bridgewater and Borad, {Mitesh J.} and Gores, {Gregory J.} and Roberts, {Lewis R.} and Marin, {Jose J.G.} and Andersen, {Jesper B.} and Domenico Alvaro and Alejandro Forner and Banales, {Jesus M.} and Vincenzo Cardinale and Macias, {Rocio I.R.} and Silve Vicent and Xin Chen and Chiara Braconi and Verstegen, {Monique M.A.} and Laura Fouassier and Alexander Scheiter and Selaru, {Florin M.} and Katja Evert and Kirsten Utpatel and Laura Broutier and Massimiliano Cadamuro and Meritxell Huch and Robert Goldin and Gradilone, {Sergio A.} and Yoshimasa Saito",

note = "Funding Information: The authors thank the European Network for the Study of Cholangiocarcinoma (ENS-CCA) and the European H2020 COST Action CA18122. The authors also acknowledge the valuable contributions of the external advisory panel. C.C. is supported by Inserm, Universit{\'e} de Rennes 1 and by a grant from the French Ministry of Health and the French National Cancer Institute (PRT-K20-136), CHU Rennes, CLCC Eug{\`e}ne Marquis, Rennes. M.M.A.V. and L.J.W.v.d.L. are supported by Medical Delta Regenerative Medicine 4D (Generating complex tissues with stem cells and printing technology) and TKI-LSH grant EMC-LSH19002. S.R. is supported by the German Research Foundation (DFG, project no. 314905040 and no. 493697503) and by German Cancer Aid (Deutsche Krebshilfe, project no. 70113922). S.V. is supported by Ministerio de Ciencia, Innovaci{\'o}n y Universidades, Convocatoria 2019 para incentivar la Incorporaci{\'o}n Estable de Doctores (IED2019-001007-I), by FEDER/Ministerio de Ciencia, Innovaci{\'o}n y Universidades – Agencia Estatal de Investigaci{\'o}n (PID2020‐116344‐RB‐100) and by the Government of Navarra-Health Research Department (58; 2018). J.V. is funded by Ministerio de Ciencia e Innovaci{\'o}n, part of Agencia Estatal de Investigaci{\'o}n (AEI), through the Retos Investigaci{\'o}n grant number PID2019-108651RJ-I00/DOI 10.13039/501100011033. The authors thank CERCA Programme/Generalitat de Catalunya for institutional support. R.I.R.M. and J.J.G.M. are supported by Instituto de Salud Carlos III, Spain (PI20/00189, PI19/00819) co-funded by the European Union. L. Fouassier belongs to a team supported by the Fondation pour la Recherche M{\'e}dicale (Equipe FRM 2020 no. EQU202003010517) and is supported by Inserm and Sorbonne Universit{\'e}, INCa and ITMO Cancer of Aviesan within the framework of the 2021–2030 Cancer Control Strategy, on funds administered by Inserm. Funding Information: C.B. receives honoraria from Incyte, Servier, BI and AstraZeneca, and research funding from Avacta and Medannex. A.F. received consultancy fees from Bayer, AstraZeneca, Roche, Boston, Exact Science and Guerbert. J.W.V. reports honoraria from Agios, AstraZeneca, Genoscience Pharma, Incyte, Mundipharma EDO, Mylan, QED, Servier, SIRTex and Zymeworks, and honoraria and non-financial support from NuCana, all outside the submitted work. The other authors declare no competing interests. Publisher Copyright: {\textcopyright} 2023, Springer Nature Limited.",

year = "2023",

month = jul,

doi = "10.1038/s41575-022-00739-y",

language = "English (US)",

volume = "20",

pages = "462--480",

journal = "Nature Reviews Gastroenterology and Hepatology",

issn = "1759-5045",

publisher = "Nature Publishing Group",

number = "7",

}

TY - JOUR

T1 - Criteria for preclinical models of cholangiocarcinoma

T2 - scientific and medical relevance

AU - CCA Model Consortium

AU - Calvisi, Diego F.

AU - Boulter, Luke

AU - Vaquero, Javier

AU - Saborowski, Anna

AU - Fabris, Luca

AU - Rodrigues, Pedro M.

AU - Coulouarn, Cédric

AU - Castro, Rui E.

AU - Segatto, Oreste

AU - Raggi, Chiara

AU - van der Laan, Luc J.W.

AU - Carpino, Guido

AU - Goeppert, Benjamin

AU - Roessler, Stephanie

AU - Kendall, Timothy J.

AU - Evert, Matthias

AU - Gonzalez-Sanchez, Ester

AU - Valle, Juan W.

AU - Vogel, Arndt

AU - Bridgewater, John

AU - Borad, Mitesh J.

AU - Gores, Gregory J.

AU - Roberts, Lewis R.

AU - Marin, Jose J.G.

AU - Andersen, Jesper B.

AU - Alvaro, Domenico

AU - Forner, Alejandro

AU - Banales, Jesus M.

AU - Cardinale, Vincenzo

AU - Macias, Rocio I.R.

AU - Vicent, Silve

AU - Chen, Xin

AU - Braconi, Chiara

AU - Verstegen, Monique M.A.

AU - Fouassier, Laura

AU - Scheiter, Alexander

AU - Selaru, Florin M.

AU - Evert, Katja

AU - Utpatel, Kirsten

AU - Broutier, Laura

AU - Cadamuro, Massimiliano

AU - Huch, Meritxell

AU - Goldin, Robert

AU - Gradilone, Sergio A.

AU - Saito, Yoshimasa

N1 - Funding Information: The authors thank the European Network for the Study of Cholangiocarcinoma (ENS-CCA) and the European H2020 COST Action CA18122. The authors also acknowledge the valuable contributions of the external advisory panel. C.C. is supported by Inserm, Université de Rennes 1 and by a grant from the French Ministry of Health and the French National Cancer Institute (PRT-K20-136), CHU Rennes, CLCC Eugène Marquis, Rennes. M.M.A.V. and L.J.W.v.d.L. are supported by Medical Delta Regenerative Medicine 4D (Generating complex tissues with stem cells and printing technology) and TKI-LSH grant EMC-LSH19002. S.R. is supported by the German Research Foundation (DFG, project no. 314905040 and no. 493697503) and by German Cancer Aid (Deutsche Krebshilfe, project no. 70113922). S.V. is supported by Ministerio de Ciencia, Innovación y Universidades, Convocatoria 2019 para incentivar la Incorporación Estable de Doctores (IED2019-001007-I), by FEDER/Ministerio de Ciencia, Innovación y Universidades – Agencia Estatal de Investigación (PID2020‐116344‐RB‐100) and by the Government of Navarra-Health Research Department (58; 2018). J.V. is funded by Ministerio de Ciencia e Innovación, part of Agencia Estatal de Investigación (AEI), through the Retos Investigación grant number PID2019-108651RJ-I00/DOI 10.13039/501100011033. The authors thank CERCA Programme/Generalitat de Catalunya for institutional support. R.I.R.M. and J.J.G.M. are supported by Instituto de Salud Carlos III, Spain (PI20/00189, PI19/00819) co-funded by the European Union. L. Fouassier belongs to a team supported by the Fondation pour la Recherche Médicale (Equipe FRM 2020 no. EQU202003010517) and is supported by Inserm and Sorbonne Université, INCa and ITMO Cancer of Aviesan within the framework of the 2021–2030 Cancer Control Strategy, on funds administered by Inserm. Funding Information: C.B. receives honoraria from Incyte, Servier, BI and AstraZeneca, and research funding from Avacta and Medannex. A.F. received consultancy fees from Bayer, AstraZeneca, Roche, Boston, Exact Science and Guerbert. J.W.V. reports honoraria from Agios, AstraZeneca, Genoscience Pharma, Incyte, Mundipharma EDO, Mylan, QED, Servier, SIRTex and Zymeworks, and honoraria and non-financial support from NuCana, all outside the submitted work. The other authors declare no competing interests. Publisher Copyright: © 2023, Springer Nature Limited.

PY - 2023/7

Y1 - 2023/7

N2 - Cholangiocarcinoma (CCA) is a rare malignancy that develops at any point along the biliary tree. CCA has a poor prognosis, its clinical management remains challenging, and effective treatments are lacking. Therefore, preclinical research is of pivotal importance and necessary to acquire a deeper understanding of CCA and improve therapeutic outcomes. Preclinical research involves developing and managing complementary experimental models, from in vitro assays using primary cells or cell lines cultured in 2D or 3D to in vivo models with engrafted material, chemically induced CCA or genetically engineered models. All are valuable tools with well-defined advantages and limitations. The choice of a preclinical model is guided by the question(s) to be addressed; ideally, results should be recapitulated in independent approaches. In this Consensus Statement, a task force of 45 experts in CCA molecular and cellular biology and clinicians, including pathologists, from ten countries provides recommendations on the minimal criteria for preclinical models to provide a uniform approach. These recommendations are based on two rounds of questionnaires completed by 35 (first round) and 45 (second round) experts to reach a consensus with 13 statements. An agreement was defined when at least 90% of the participants voting anonymously agreed with a statement. The ultimate goal was to transfer basic laboratory research to the clinics through increased disease understanding and to develop clinical biomarkers and innovative therapies for patients with CCA.

AB - Cholangiocarcinoma (CCA) is a rare malignancy that develops at any point along the biliary tree. CCA has a poor prognosis, its clinical management remains challenging, and effective treatments are lacking. Therefore, preclinical research is of pivotal importance and necessary to acquire a deeper understanding of CCA and improve therapeutic outcomes. Preclinical research involves developing and managing complementary experimental models, from in vitro assays using primary cells or cell lines cultured in 2D or 3D to in vivo models with engrafted material, chemically induced CCA or genetically engineered models. All are valuable tools with well-defined advantages and limitations. The choice of a preclinical model is guided by the question(s) to be addressed; ideally, results should be recapitulated in independent approaches. In this Consensus Statement, a task force of 45 experts in CCA molecular and cellular biology and clinicians, including pathologists, from ten countries provides recommendations on the minimal criteria for preclinical models to provide a uniform approach. These recommendations are based on two rounds of questionnaires completed by 35 (first round) and 45 (second round) experts to reach a consensus with 13 statements. An agreement was defined when at least 90% of the participants voting anonymously agreed with a statement. The ultimate goal was to transfer basic laboratory research to the clinics through increased disease understanding and to develop clinical biomarkers and innovative therapies for patients with CCA.

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U2 - 10.1038/s41575-022-00739-y

DO - 10.1038/s41575-022-00739-y

M3 - Article

C2 - 36755084

AN - SCOPUS:85147649578

SN - 1759-5045

VL - 20

SP - 462

EP - 480

JO - Nature Reviews Gastroenterology and Hepatology

JF - Nature Reviews Gastroenterology and Hepatology

IS - 7

ER -

Criteria for preclinical models of cholangiocarcinoma: scientific and medical relevance

Abstract

Bibliographical note

PubMed: MeSH publication types

UN SDGs

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