Targeted therapies in pediatric oncology: A start

The treatment of cancers was revolutionized by the use of combination "conventional" chemotherapy, first in children, during the 20^th century. That foundation of therapy, while having achieved significant advances in survival, has reached its limits. Innovations in targeted therapies are now building upon that foundation to further improve outcomes in pediatric oncology. These targeted agents are designed to narrowly utilize specific biologic processes for greater efficacy and optimally fewer generalized toxicities. I will provide an overview of the current spectrum of targeted therapies available against childhood cancers, categorized into immunotherapies and molecular targeted therapies. I review approved drugs within each class of agents as well as drugs that have entered early phase trials, either in the past or at present. I then conclude with a brief discussion of current challenges and next steps to the further advancement of new targeted therapies for pediatric malignancies.