Preclinical models of childhood cancer for the development of targeted therapies

Childhood cancer represents a diverse group of malignancies that overall constitute approximately one percent of human cancer. With current multimodality therapies comprising surgery, radiation therapy and intensive chemotherapy, about seventy percent of children are cured of disease, and the 5-year Event-Free survival is approaching 80%. However, gains from current cytotoxic therapies come with a severe cost to the health of survivors. These rare cancers present unique challenges to developing new therapies, in part because relatively few clinical trials can be undertaken. Hence, developing preclinical models that accurately predict responsiveness to new agents assumes an increased importance. Here we review recent advances in preclinical models for development of molecularly targeted therapies.