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@ARTICLE{Vassal:169158,
      author       = {G. Vassal and P. J. Houghton and S. M. Pfister$^*$ and M.
                      A. Smith and H. N. Caron and X.-N. Li and D. J. Shields and
                      O. Witt$^*$ and J. J. Molenaar and S. Colombetti and J.
                      Schuler and L. F. Stancato},
      title        = {{I}nternational consensus on minimum preclinical testing
                      requirements for the development of innovative therapies for
                      children and adolescents with cancer.},
      journal      = {Molecular cancer therapeutics},
      volume       = {20},
      number       = {8},
      issn         = {1538-8514},
      address      = {Philadelphia, Pa.},
      publisher    = {AACR},
      reportid     = {DKFZ-2021-01292},
      pages        = {1462-1468},
      year         = {2021},
      note         = {2021 Aug;20(8):1462-1468},
      abstract     = {Cancer remains the leading cause of disease-related death
                      in children. For the many children who experience relapses
                      of their malignant solid tumors, usually after very
                      intensive first-line therapy, curative treatment options are
                      scarce. Preclinical drug testing to identify promising
                      treatment elements that match the molecular make-up of the
                      tumor is hampered by the fact that 1) molecular genetic data
                      on pediatric solid tumors from relapsed patients and thus
                      our understanding of tumor evolution and therapy resistance
                      are very limited to date and 2) for many of the high-risk
                      entities, no appropriate and molecularly well characterized
                      patient-derived models and/or genetic mouse models are
                      currently available. However, recent regulatory changes
                      enacted by the EMA (class waiver changes) and the maturation
                      of the RACE for Children act with the FDA, will require a
                      significant increase in preclinical pediatric cancer
                      research and clinical development must occur. We detail the
                      outcome of a pediatric cancer international multistakeholder
                      meeting whose output aims at defining an international
                      consensus on minimum preclinical testing requirements for
                      the development of innovative therapies for children and
                      adolescents with cancer. Recommendations based on the
                      experience of the NCI funded PPTP/C (www.ncipptc.org) and
                      the EU funded ITCC-P4 public private partnership
                      (www.itccp4.eu) are provided for the use of cell-based and
                      mouse models for pediatric solid malignancies, as well as
                      guidance on the scope and content of preclinical
                      proof-of-concept data packages to inform clinical
                      development dependent on clinical urgency. These
                      recommendations can serve as a minimal guidance necessary to
                      jumpstart preclinical pediatric research globally.},
      cin          = {B062 / HD01 / B310},
      ddc          = {570},
      cid          = {I:(DE-He78)B062-20160331 / I:(DE-He78)HD01-20160331 /
                      I:(DE-He78)B310-20160331},
      pnm          = {312 - Funktionelle und strukturelle Genomforschung
                      (POF4-312)},
      pid          = {G:(DE-HGF)POF4-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:34108262},
      doi          = {10.1158/1535-7163.MCT-20-0394},
      url          = {https://inrepo02.dkfz.de/record/169158},
}