Session Speaker

Targeting Tots: Challenges in Pediatric Cancer Drug Development
Jessica Boklan
USA

Growing knowledge of tumor biology has led to the emergence of promising new agents that target molecular pathways involved in oncogenesis, heralding an exciting new era in cancer drug development. This targeted approach, however, adds yet more challenges to an already long list of issues being faced in drug development for a small subset of cancer patients - children. While the overall cure rate for childhood cancer approaches 75%, cancer is still the most common cause of death from disease in U.S children. The many issues facing pediatric drug development today include problems accessing new agents, financial priorities, differences between adult and pediatric tumor biology, and small patient numbers. The latter becomes especially problematic as patients are not only sub grouped by disease, but increasingly by molecular classification. As a result, the numbers of patients in each group grows smaller and smaller. Difficult decisions must be made regarding which of the many targeted agents to bring forward into pediatric trials and how to design those trials to maximize efficiency and the use of available resources, including patients. We will discuss these challenges and some approaches being taken to address them.