The ITCC-P4 PDX platform of pediatric cancers for preclinical testing
Federico, A.; Gopisetty, A.; Surdez, D.; Iddir, Y.; Autry, R. J.; Waterfall, J.; Saberi-Ansari, E.; Bobin, C.; Ballet, S.; Pierron, G.; Wierzbinska, J.; Schlicker, A.; Sill, M.; Volckmann, R.; Zwijnenburg, D. A.; Mackay, A.; Zaidi, S.; Saint-Charles, A.; Mack, N.; Schwalm, B.; Weiser, L.; Buchhalter, I.; Previti, C.; Boettcher, A.-L.; Iradier, F.; Rief, E.-M.; Jones, D. T. W.; Witt, O.; Westermann, F.; Milde, T.; Eggert, A.; Huebener, N.; Schulte, J.; Colombetti, S.; Chesler, L.; Kovar, H.; Klusmann, J.-H.; Debatin, K.-M.; Bomken, S.; Guttke, C.; Hamerlik, P.; Hattersley, M.; Garcia, M.; Colla
Show 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 in order to improve pediatric cancer patients survival. Here, we report the EU-IMI-2 funded public-private partnership "ITCC-Pediatric Preclinical Proof-of-Concept Platform" (ITCC-P4), which has built a large repertoire of patient-derived xenograft (PDX) models, representing all major solid pediatric cancer types, for in vivo drug testing. Three-hundred-fifty-three PDX models from diagnostic and relapsed pediatric cancers have been established and molecularly characterized, together with matched germline/tumor samples. As proof-of-concept, we present in vivo drug screening data in neuroblastoma and rhabdomyosarcoma models. PDX data, accessible at http://r2platform.com/itcc-p4, allow the selection of models based on oncogenic drivers and/or potential biomarkers for preclinical testing. Operated by a non-profit entity (www.itccp4.com), this sustainable platform aids academic and industrial researchers in developing and prioritizing innovative therapies for pediatric cancer. Graphical abstract O_FIG O_LINKSMALLFIG WIDTH=200 HEIGHT=112 SRC="FIGDIR/small/703023v1_ufig1.gif" ALT="Figure 1"> View larger version (41K): org.highwire.dtl.DTLVardef@195ba30org.highwire.dtl.DTLVardef@f2c2d9org.highwire.dtl.DTLVardef@1d63f4dorg.highwire.dtl.DTLVardef@d60027_HPS_FORMAT_FIGEXP M_FIG C_FIG
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