New approaches to therapeutic drug development for childhood cancers.

ABSTRACT

PURPOSE OF REVIEW We provide an overview of the current landscape of drug development relevant to childhood cancers. We present recent and ongoing efforts to identify therapeutic targets in pediatric cancers. We describe efforts to improve the approach to clinical trials and highlight the role regulatory changes and multistakeholder platforms play in advancing pediatric cancer drug development. RECENT FINDINGS: Expanding knowledge of the genetic landscape of pediatric malignancies through clinical genomics studies has yielded an increasing number of potential targets for intervention. In parallel, new therapies for children with cancer have shifted from cytotoxic agents to targeted therapy, with examples of striking activity in patients with tumors driven by oncogenic kinase fusions. Innovative trial designs and recent governmental policies provide opportunities for accelerating development of targeted therapies in pediatric oncology. SUMMARY: Novel treatment strategies in pediatric oncology increasingly utilize molecularly targeted agents either as monotherapy or in combination with conventional cytotoxic agents. The interplay between new target identification, efforts to improve clinical trial design and new government regulations relevant to pediatric cancer drug development has the potential to advance novel agents into frontline care of children with cancer.