Coming from the world of families with children ravaged by the many rare and catastrophic epilepsies, we suggest consideration of a benchmark where every child with epilepsy is enrolled in a clinical trial. Our children are already guinea pigs, but their experiences are not treated with the respect of a common lab rat. The vital data on the impact of the constantly changing cocktails to try to control their treatment resistant epilepsy is never captured, archived, combined, or accessible to either clinicians or researchers. So basic but vital questions remain mysteries: What works and for whom and under what circumstances? What treatments were toxic or even life-threatening for some children and why? The drugs their bodies are pumped full of were never tested in children – and yet we fail to capitalize on the potential to learn from every precious opportunity in their constantly evolving treatment and outcomes.
There are two nascent "epilepsy learning health systems" trying to set up the infrastructure to begin to do this, but they are woefully underfunded, limiting their development, application, reach and effectiveness. Yet the experience in pediatric oncology over many decades has fully demonstrated the enormous payoffs of integrating research into clinical care by enrolling nearly every child in some form of clinical research trial. Such an undertaking would not only fuel and empower more robust research on factors affecting the efficacy of widely varying treatments, but the learnings would be constantly applied, accelerating meaningful improvements in the quality of our children's lives.
It is clear full support for such an undertaking – enrolling every child with epilepsy in clinical trials - is well beyond the current funding capacity of NIH. However, surely there are important and effective ways NIH, perhaps in collaboration with FDA and CDC, can immediately dedicate seed research funding to this endeavor to test and likely demonstrate enormously high payoffs in expanded knowledge and improved outcomes sufficient to garner legislative support for a long overdue Epilepsy Moonshot: Curing the Epilepsies!