A biobank of pediatric patient-derived-xenograft models in cancer precision medicine trial MAPPYACTS for relapsed and refractory tumors

Pediatric patients with recurrent and refractory cancers are in most need for new treatments. This study developed patient-derived-xenograft (PDX) models within the European MAPPYACTS cancer precision medicine trial (NCT02613962). To date, 131 PDX models were established following heterotopical and/...

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Detalhes bibliográficos
Autores: Marques da Costa, Maria Eugénia, Zaidi, Sakina, Scoazec, Jean Yves, Droit, Robin, Lim, Wan Ching, Marchais, Antonin, Salmon, Jerome, Cherkaoui, Sarah, Morscher, Raphael J., Laurent, Anouchka, Malinge, Sébastien, Mercher, Thomas, Tabone Eglinger, Séverine, Goddard, Isabelle, Pflumio, Francoise, Calvo, Julien, Redini, Francoise, Entz-Werlé, Natacha, Soriano, Aroa, Villanueva, Alberto, Cairo, Stefano, Chastagner, Pascal, Moro, Massimo, Owens, Cormac, Casanova, Michela, Hladun Alvaro, Raquel, Berlanga, Pablo, Daudigeos Dubus, Estelle, Dessen, Philippe, Zitvogel, Laurence, Lacroix, Ludovic, Pierron, Gaelle, Delattre, Olivier, Schleiermacher, Gudrun, Surdez, Didier, Geoerger, Birgit
Formato: artículo
Estado:Versión publicada
Fecha de publicación:2023
País:España
Recursos:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
Repositorio:Recercat. Dipósit de la Recerca de Catalunya
OAI Identifier:oai:recercat.cat:2445/204382
Acesso em linha:https://hdl.handle.net/2445/204382
Access Level:acceso abierto
Palavra-chave:Càncer
Pediatria
Cancer
Pediatrics
Descrição
Resumo:Pediatric patients with recurrent and refractory cancers are in most need for new treatments. This study developed patient-derived-xenograft (PDX) models within the European MAPPYACTS cancer precision medicine trial (NCT02613962). To date, 131 PDX models were established following heterotopical and/or orthotopical implantation in immunocompromised mice: 76 sarcomas, 25 other solid tumors, 12 central nervous system tumors, 15 acute leukemias, and 3 lymphomas. PDX establishment rate was 43%. Histology, whole exome and RNA sequencing revealed a high concordance with the primary patient's tumor profile, human leukocyte-antigen characteristics and specific metabolic pathway signatures. A detailed patient molecular characterization, including specific mutations prioritized in the clinical molecular tumor boards are provided. Ninety models were shared with the IMI2 ITCC Pediatric Preclinical Proof-of-concept Platform (IMI2 ITCC-P4) for further exploitation. This PDX biobank of unique recurrent childhood cancers provides an essential support for basic and translational research and treatments development in advanced pediatric malignancies.

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