ITCC P4 - PAEDIATRIC PRECLINICAL PROOF OF CONCEPT PLATFORM
Cancer remains the leading cause of disease-related death in children. For the ~25% of 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 options that match the molecular make-up of the tumor is hampered by the fact that
- 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
- for many of the high-risk entities, no appropriate and molecularly well characterized patient-derived models are currently available.
Thus, quality-assured upfront preclinical testing of novel molecularly targeted compounds in a repertoire of well-characterized models will establish the basis to increase therapeutic successes of these drugs in children with solid malignancies. Since these tumors are overall genetically much less complex than their adult counterparts, it is anticipated that it will be easier to identify powerful predictive biomarkers to allow for accurate matching of targets and drugs.