TY - JOUR
T1 - A biobank of pediatric patient-derived-xenograft models in cancer precision medicine trial MAPPYACTS for relapsed and refractory tumors
AU - Marques Da Costa, Maria Eugénia
AU - Zaidi, Sakina
AU - Scoazec, Jean Yves
AU - Droit, Robin
AU - Lim, Wan Ching
AU - Marchais, Antonin
AU - Salmon, Jerome
AU - Cherkaoui, Sarah
AU - Morscher, Raphael J.
AU - Laurent, Anouchka
AU - Malinge, Sébastien
AU - Mercher, Thomas
AU - Tabone-Eglinger, Séverine
AU - Goddard, Isabelle
AU - Pflumio, Francoise
AU - Calvo, Julien
AU - Redini, Francoise
AU - Entz-Werlé, Natacha
AU - Soriano, Aroa
AU - Villanueva, Alberto
AU - Cairo, Stefano
AU - Chastagner, Pascal
AU - Moro, Massimo
AU - Owens, Cormac
AU - Casanova, Michela
AU - Hladun-Alvaro, Raquel
AU - Berlanga, Pablo
AU - Daudigeos-Dubus, Estelle
AU - Dessen, Philippe
AU - Zitvogel, Laurence
AU - Lacroix, Ludovic
AU - Pierron, Gaelle
AU - Delattre, Olivier
AU - Schleiermacher, Gudrun
AU - Surdez, Didier
AU - Geoerger, Birgit
N1 - Publisher Copyright:
© 2023, Springer Nature Limited.
PY - 2023/12/1
Y1 - 2023/12/1
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85171577476&partnerID=8YFLogxK
U2 - 10.1038/s42003-023-05320-0
DO - 10.1038/s42003-023-05320-0
M3 - Article
C2 - 37723198
AN - SCOPUS:85171577476
SN - 2399-3642
VL - 6
JO - Communications Biology
JF - Communications Biology
IS - 1
M1 - 949
ER -