Statement of Need
Pediatric oncology has come a long was in the last several decades. Cure rates have improved overall and advances in research have provided seminal insights into the cancer problem in general.
However, this cure rate has come at significant cost. First, the intensity of therapies used to has increased substantially, so the majority of childhood cancer patients have significant long-term complications.
Clearly, cure is not enough. Of equal urgency, several cancers, especially the solid malignancies, have not shown improvement in cure rates and current treatments are at their maximal intensity. Indeed, several cancers have seen no significant progress in over a decade and childhood cancer remains the number one cause of death in children from disease (accidents claim the most lives).
Cancer research is responsible for the achievements to date, with pediatric oncologist showing the world how to perform meaningful clinical research that influences clinical care. However, the next generation of research that will lead to meaningful differences is quite different, and will rely on a detailed understanding of cancer biology with a newly facile was to translate discoveries to the clinic. It is often said that it takes a decade to develop a new cancer drug.
Clearly, this is not acceptable. We have dramatically decreased the timeline to get new therapies to children with refractory cancer, and this new translational molecular oncology will be the pathway to the next revolution in cure rates. However, this type of research is resource intensive.
Philanthropic investment will provide the essential catalyst to make more discoveries and move them to improving cure rates as quickly as possible, and a partnership between researchers and "investors" in this mission will be critical to expand in order to make a difference now.
John M. Maris, MD, Children's Hospital of Philadelphia