ABSTRACT

The popular adage “children are not small adults” represents a double-edged sword when it comes to identifying new medicines to treat childhood cancer: while children are recognized in their own rights and needs, the adage also calls attention to the fact that therapeutic approaches established for adults may not readily extend to children. Even though childhood cancer is a relatively rare diagnosis, it is estimated that 1 in 285 children in the United States will be diagnosed with the disease before the age of 20 years [1]. In contrast, the lifetime risk of developing cancer is approximately one in two for men and one in three for women, where 78% of all cancer diagnoses are in people aged 55 years or older [1]. Pediatric cancers represent 1% of all new cancer diagnoses in the United States [1]. On an annual basis, these rates are estimated to result in 10,450 new cases and 1,350 cancer deaths among U.S. children (aged 0–14 years) and 5,330 new cases and 610 cancer deaths among U.S. adolescents (aged 15–19 years) [1]. The estimated number of annual new diagnoses of cancer in children worldwide is 175,000. In contrast, approximately 1.7 million new adult cancer cases are expected to be diagnosed during 2015 in the United States alone. Nevertheless, cancer is the most common cause of nonviolent death for children in the United States, and it remains a significant area of focus for treatment [2].