Concept and approach

The overall concept of the proposal involves all critical steps that are necessary to achieve the primary goal of this project, namely the establishment of a sustainable comprehensive preclinical pediatric testing platform based on a panel of ~420 well-characterized patient-derived models of high-risk pediatrictumors to be used for rational pediatric drug development. We hypothesize that the exact matching of a validated target with a novel drug, demonstrated by high-quality preclinical testing in faithful patient-derived disease models, will improve patient stratification for novel treatment options and ultimately patient outcome. As specified above, our highly inter-disciplinary, multi-national team is ideally suited to successfully tackle the challenges of this topic and, in the true spirit of IMI2, tremendously help in translating patient-oriented research for high-risk patients into clinical application.