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000294424 0247_ $$2ISSN$$a0099-7374
000294424 0247_ $$2ISSN$$a0008-5472
000294424 0247_ $$2ISSN$$a1538-7445
000294424 037__ $$aDKFZ-2024-02253
000294424 082__ $$a610
000294424 1001_ $$aFederico, Aniello$$b0
000294424 245__ $$aAbstract 3571: The ITCC-P4 sustainable platform of fully characterized PDXs supports the preclinical proof-of-concept drug testing of high-risk pediatric tumor models
000294424 260__ $$c2023
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000294424 520__ $$aCancer represents a leading cause of death by disease in childhood. Pediatric tumors exhibit a high intertumoral heterogeneity, as different tumor types and subtypes have emerged with peculiar molecular and clinical features; however, compared to cancer in adults, pediatric tumors are rare and mostly present with lower mutational burden. The lack of specific therapeutic options represents the main current challenge; systematic, multi-disciplinary approaches are required to accelerate drug development and ultimately to find cures for all children with cancer. The EU funded “Innovative Therapies for Children with Cancer–Pediatric Preclinical Proof-of-Concept Project” (ITCC-P4; www.itccp4.eu) consortium consists of a public-private partnership including academic and industrial partners with the goal of developing a large-scale platform comprising >400 patient-derived xenograft (PDX) models representing high-risk pediatric cancers. Currently, this collection of PDX models includes the most common types of pediatric tumors, such as leukemia (n=28), bone and soft-tissue sarcomas (n=154), CNS tumors (n=96) and neuroblastomas (n=38), as well as other rare childhood cancers, such as hepatoblastomas (n=20) and malignant rhabdoid tumors (n=18); PDX models have been generated either from primary (n=206) or relapse (n=118) disease. In order to: a) investigate the biology of the pediatric PDX models in a high-throughput and systematic fashion, b) assess whether they accurately reflect the molecular features of the corresponding primary tumor and, c) identify potential new suitable biomarkers, we performed a comprehensive molecular characterization (whole-exome and low-coverage whole-genome sequencing; DNA methylation profiling; RNAseq and gene expression profiling) of the PDX models, as well as their matching human tumors and germline samples. These data contributed to the stratification of the PDX models based on their mutational status and emerging molecular vulnerabilities to inform in vivo drug testing in all these PDX models. This proof-of-concept drug testing has been conducted defining, for each group of models, a panel of single compounds (SOC n=3; novel targeted therapies, n=6) or combinations (with each other or with chemo- or radiotherapy). All processed molecular and drug-testing data are collected in the consortium´s centralized data repository (https://r2.amc.nl) allowing data downstream analysis, visualization and interpretation. Taken together, the ITCC-P4 sustainable platform represents a validated and powerful tool to investigate the biology of pediatric cancer based on the establishment, characterization and preclinical testing of pediatric cancer PDX models, ultimately envisaged to contribute the development of innovative therapeutic options for childhood cancer patients.
000294424 588__ $$aDataset connected to CrossRef, Journals: inrepo02.dkfz.de
000294424 7001_ $$aGopisetty, Apurva$$b1
000294424 7001_ $$aSurdez, Didier$$b2
000294424 7001_ $$aIddir, Yasmine$$b3
000294424 7001_ $$aSaint-Charles, Alexandra$$b4
000294424 7001_ $$aWierzbinska, Justyna$$b5
000294424 7001_ $$aSchlicker, Andreas$$b6
000294424 7001_ $$aVolckmann, Richard$$b7
000294424 7001_ $$aZwijnenburg, Danny$$b8
000294424 7001_ $$aColombetti, Sara$$b9
000294424 7001_ $$aHeidenreich, Olaf$$b10
000294424 7001_ $$aIradier, Fatima$$b11
000294424 7001_ $$aKovar, Heinrich$$b12
000294424 7001_ $$aKlusmann, Jan-Henning$$b13
000294424 7001_ $$aDebatin, Klaus-Michael$$b14
000294424 7001_ $$aBomken, Simon$$b15
000294424 7001_ $$aGuttke, Christina$$b16
000294424 7001_ $$aHattersley, Maureen M.$$b17
000294424 7001_ $$aColland, Frédéric$$b18
000294424 7001_ $$aStrougo, Ashley$$b19
000294424 7001_ $$aGuillén, María José$$b20
000294424 7001_ $$aChesler, Louis$$b21
000294424 7001_ $$aJones, Chris$$b22
000294424 7001_ $$ada Costa, Maria Eugénia Marques$$b23
000294424 7001_ $$aScotlandi, Katia$$b24
000294424 7001_ $$aMoro, Massimo$$b25
000294424 7001_ $$aSchäfer, Beat$$b26
000294424 7001_ $$aWachtel, Marco$$b27
000294424 7001_ $$aGojo, Johannes$$b28
000294424 7001_ $$aBerger, Walter$$b29
000294424 7001_ $$aCarcaboso, Ángel Montero$$b30
000294424 7001_ $$aGürgen, Dennis$$b31
000294424 7001_ $$aHoffmann, Jens$$b32
000294424 7001_ $$aIndersie, Emilie$$b33
000294424 7001_ $$aCairo, Stefano$$b34
000294424 7001_ $$aSchueler, Julia$$b35
000294424 7001_ $$aHuebener, Nicole$$b36
000294424 7001_ $$aSchulte, Johannes H.$$b37
000294424 7001_ $$aMolenaar, Jan J.$$b38
000294424 7001_ $$aGeoerger, Birgit$$b39
000294424 7001_ $$aShields, David J.$$b40
000294424 7001_ $$aCaron, Hubert N.$$b41
000294424 7001_ $$aVassal, Gilles$$b42
000294424 7001_ $$aStancato, Lou F.$$b43
000294424 7001_ $$aStancato, Lou F.$$b44
000294424 7001_ $$aPfister, Stefan M.$$b45
000294424 7001_ $$aJäger, Natalie$$b46
000294424 7001_ $$aKoster, Jan$$b47
000294424 7001_ $$aKool, Marcel$$b48
000294424 7001_ $$aSchleiermacher, Gudrun$$b49
000294424 773__ $$0PERI:(DE-600)2036785-5$$a10.1158/1538-7445.AM2023-3571$$gVol. 83, no. 7_Supplement, p. 3571 - 3571$$x1538-7445$$y2023
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