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The ITCC-P4 PDX Platform Enables Preclinical Testing of Pediatric Cancers

Lookup NU author(s): Dr Simon BomkenORCiD

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This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).

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.

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Author(s): Federico A, Gopisetty A, Surdez D, Iddir Y, Autry RJ, Waterfall JJ, Saberi-Ansari E, Bobin C, Ballet S, Pierron G, Wierzbinska JA, Schlicker A, Sill M, Volckmann R, Zwijnenburg DA, Mackay A, Zaïdi S, Saint-Charles A, Mack N, Schwalm B, Brabetz S, Weiser L, Buchhalter I, Previti C, Böttcher AL, Iradier F, Rief EM, Jones DTW, Witt O, Westermann F, Milde T, Eggert A, Huebener N, Schulte JH, Colombetti S, Chesler L, Tucker ER, Morcavallo A, Lorenzi F, Kovar H, Klusmann JH, Debatin KM, Bomken S, Guttke CD, Hamerlik P, Hattersley MM, Garcia M, Colland F, Strougo A, Aviles P, Zapotocky M, Sumerauer D, Gojo J, Berger W, Lötsch-Gojo D, Schueler J, Girard EJ, Olson JM, Schäfer BW, Wachtel M, Molenaar JJ, Dumevska B, Fernandez Potente E, Jones C, Carcaboso AM, Indersie E, Cairo S, Scotlandi K, Manara MC, Landuzzi L, Di Giannatale A, Gasparini P, Moro M, Gürgen D, Hoffmann J, Marques da Costa ME, Geoerger B, Delattre O, Shields DJ, Caron HN, Vassal G, Stancato LF, Pfister SM, Jäger N, Koster J, Kool M, Schleiermacher G

Publication type: Article

Publication status: Published

Journal: Cancer Research

Year: 2026

Pages: epub ahead of print

Online publication date: 30/07/2026

Acceptance date: 09/07/2026

Date deposited: 07/09/2026

ISSN (print): 0008-5472

ISSN (electronic): 1538-7445

Publisher: American Association for Cancer Research

URL: https://doi.org/10.1158/0008-5472.CAN-25-5295

DOI: 10.1158/0008-5472.CAN-25-5295

ePrints DOI: 10.57711/fqcb-ss45

Data Access Statement: The curated multiomics data are accessible via the R2 ITCC-P4 PDX Datascope portal (http://r2platform.com/itcc-p4/). Raw sequencing data are accessible via the European Genome-phenome Archive, access number: EGAS00001008330). Any other data shown in this study are available upon request. For inquiries about the data, please contact ITCC-P4 gGmbH (info@itccp4.com).

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