History of Pediatric Cancer Clinical Research

In 1971 president Richard Nixon declared a war on cancer and announced his goal to cure cancer by 1976, the bicentennial year. Thirty nine years and more than 100 billion dollars later, the cumulative adult death rate from cancer adjusted for the size and age of the population has improved by less than 5% [1]. In comparison, the death rate for heart disease over this time period has improved by more than 64% [1]. In 2009, the NY Times published a series on the “war on cancer” specifically highlighting some of the suspected causes for these disappointing results. The primary aim of the series was to investigate and discuss the translational research efforts over the past several decades and to explore some of the strategic decisions made by funding agencies as it relates to basic science and clinical research in order to move new therapies into the clinic quickly and safely. There is no simple explanation for why the death rate due to cancer hasn’t improved more than 5% over the past 4 decades. However, the progress made in treating pediatric cancer over the same time period may shed some light on ways to improve our approach to translational research in coming years. Today, the overall cure rate for pediatric cancers approaches 80%; this is a 30% improvement since 1971. This is remarkable when we consider the rarity of pediatric cancer, the limited research funding and lack of investment by the pharmaceutical industry. Most of the progress in improving outcome for pediatric cancer has come from clinical research. Indeed, the majority (>90%) of pediatric cancer patients are enrolled on treatment protocols and there is now abundant evidence that research protocols have helped optimize treatment intensification, drug dosing and timing, chemotherapeutic drug combination, and the identification of prognostic features of disease in relation to treatment plans. In sharp contrast, only 3% of adult cancer patients are enrolled on research protocols [2]. These numbers suggest that the advances in patient outcome for pediatric cancer since the beginning of the war on cancer can be attributed in part to the coordinated participation in clinical research protocols.