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@INPROCEEDINGS{Federico:294424,
      author       = {A. Federico and A. Gopisetty and D. Surdez and Y. Iddir and
                      A. Saint-Charles and J. Wierzbinska and A. Schlicker and R.
                      Volckmann and D. Zwijnenburg and S. Colombetti and O.
                      Heidenreich and F. Iradier and H. Kovar and J.-H. Klusmann
                      and K.-M. Debatin and S. Bomken and C. Guttke and M. M.
                      Hattersley and F. Colland and A. Strougo and M. J. Guillén
                      and L. Chesler and C. Jones and M. E. M. da Costa and K.
                      Scotlandi and M. Moro and B. Schäfer and M. Wachtel and J.
                      Gojo and W. Berger and Á. M. Carcaboso and D. Gürgen and
                      J. Hoffmann and E. Indersie and S. Cairo and J. Schueler and
                      N. Huebener and J. H. Schulte and J. J. Molenaar and B.
                      Geoerger and D. J. Shields and H. N. Caron and G. Vassal and
                      L. F. Stancato and L. F. Stancato and S. M. Pfister and N.
                      Jäger and J. Koster and M. Kool and G. Schleiermacher},
      title        = {{A}bstract 3571: {T}he {ITCC}-{P}4 sustainable platform of
                      fully characterized {PDX}s supports the preclinical
                      proof-of-concept drug testing of high-risk pediatric tumor
                      models},
      issn         = {1538-7445},
      reportid     = {DKFZ-2024-02253},
      year         = {2023},
      abstract     = {Cancer 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.},
      ddc          = {610},
      typ          = {PUB:(DE-HGF)1},
      doi          = {10.1158/1538-7445.AM2023-3571},
      url          = {https://inrepo02.dkfz.de/record/294424},
}