학술논문
A biobank of pediatric patient-derived-xenograft models in cancer precision medicine trial MAPPYACTS for relapsed and refractory tumors.
Document Type
Article
Author
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
Source
Subject
*INDIVIDUALIZED medicine
*CHILD patients
CENTRAL nervous system tumors
*
Language
ISSN
2399-3642
Abstract
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. A collection of 131 multi-panel pediatric tumor PDX models are generated and characterized in a comprehensive effort to provide support for basic and translational research and treatment development in advanced pediatric malignancies. [ABSTRACT FROM AUTHOR]