New approaches to drug development in pediatric oncology.

This article reviews the changing paradigm of new drug development for childhood cancer. Although there have been dramatic improvements in the treatment and outcome for childhood cancer over the past 50 years, in some cancers, only limited progress has been made. New strategies to improve the outcome for poor-prognosis tumors and to minimize the long-term sequelae associated with therapy are required. Such strategies include a new pediatric-specific initiative for the preclinical testing of anticancer drugs for childhood malignancies, and ongoing research into the molecular heterogeneity of childhood tumors to facilitate the evaluation of molecularly targeted drugs. In addition, continued investigation into the genetic basis underlying interpatient variability in drug exposure and response is a priority. The goal of these initiatives is to improve the overall outcome for childhood cancer while minimizing the short and long-term sequelae associated with current treatment strategies.