Authors
Aniello Federico, Apurva Gopisetty, Didier Surdez, Yasmine Iddir, Robert J Autry, Joshua J Waterfall, Elnaz Saberi-Ansari, Charles Bobin, Stelly Ballet, Gaelle Pierron, Justyna A Wierzbinska, Andreas Schlicker, Martin Sill, Richard Volckmann, Danny A Zwijnenburg, Alan Mackay, Sakina Zaïdi, Alexandra Saint-Charles, Norman Mack, Benjamin Schwalm, Sebastian Brabetz, Lena Weiser, Ivo Buchhalter, Christopher Previti, Anna-Lisa Böttcher, Fatima Iradier, Eva-Maria Rief, David T W Jones, Olaf Witt, Frank Westermann, Till Milde, Angelika Eggert, Nicole Huebener, Johannes H Schulte, Sara Colombetti, Louis Chesler, Elizabeth R Tucker, Alaide Morcavallo, Federica Lorenzi, Heinrich Kovar, Jan-Henning Klusmann, Klaus-Michael Debatin, Simon Bomken, Christina D Guttke, Petra Hamerlik, Maureen M Hattersley, Michelle Garcia, Frédéric Colland, Ashley Strougo, Pablo Aviles, Michal Zapotocky, David Sumerauer, Johannes Gojo, Walter Berger, Daniela Lötsch-Gojo, Julia Schueler, Emily J Girard, James M Olson, Beat W Schäfer, Marco Wachtel, Jan J Molenaar, Biljana Dumevska, Elisabet Fernandez Potente, Chris Jones, Angel M Carcaboso, Emilie Indersie, Stefano Cairo, Katia Scotlandi, Maria Cristina Manara, Lorena Landuzzi, Angela Di Giannatale, Patrizia Gasparini, Massimo Moro, Dennis Gürgen, Jens Hoffmann, Maria Eugenia Marques da Costa, Birgit Geoerger, Olivier Delattre, David J Shields, Hubert N Caron, Gilles Vassal, Louis F Stancato, Stefan M Pfister, Natalie Jäger, Jan Koster, Marcel Kool, Gudrun Schleiermacher
Published in
Cancer research. Jul 10, 2026. Epub Jul 10, 2026.
Abstract
Cancer is the leading cause of disease-related deaths among children in high-income countries. Tumor heterogeneity and lack of mechanism-of-action-based therapeutic options are key challenges to overcome to improve pediatric cancer patient survival. To address these challenges, we formed the EU-IMI-2 funded public-private partnership "ITCC-Pediatric Preclinical Proof-of-Concept Platform" (ITCC-P4), which built a large repertoire of patient-derived xenograft (PDX) models representing all major high-risk solid pediatric cancer types for in vivo drug testing. A total of 353 PDX models were established from diagnostic and relapsed pediatric cancers and molecularly characterized, together with matched germline/tumor samples. Serial PDX models were also established, spanning diagnostic/posttreatment, primary/relapse, and metastasis-derived pairs. Proof-of-concept in vivo drug screening data in neuroblastoma and rhabdomyosarcoma models identified potential predictive biomarkers for targeted therapy. Molecular data from the PDX models, accessible at https://r2platform.com/itcc-p4, allowed the selection of models for preclinical testing based on oncogenic drivers and/or potential biomarkers. Operated by a non-profit entity, this sustainable platform aids academic and industrial researchers in developing and prioritizing innovative therapies for pediatric cancer.
PMID:
42430560
Bibliographic data and abstract were imported from PubMed on 11 Jul 2026.
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