Fig. 1.

Improving therapy of pediatric cancers through collaboration. Next-generation sequencing of tumor samples may directly identify candidate targeted therapies. In many cases, further investigation is required. Model systems such as organoids or genetically engineered animals can interrogate the function of candidate driver genes in a setting that recapitulates the complexity of the in vivo tumor environment. Such models can support drug screening and preclinical testing of novel therapies. Throughout, collaboration between clinicians and basic scientists is essential to define clinical challenges and to build and refine disease models.