Date:  January 11-13, 2013

Subject:  The Best Next Steps To Advance The Field Of Pediatric Neurology

Location:  La Jolla, CA

Attendees:  Top scientists and researchers including: Jeffrey Macklis, William Mobley, Mark Noble, Evan Snyder, Gary Clark, Stephen Huhn, Jeanne Loring, Margot Mayer-Proschel, Robert “Willie” Mays, Sidhartha Tan, and Alysson Muotri.  As well as parent advocates, Bernard Siegel of Genetic Policy Institute, Pediatric Brain Foundation staff and interns, and CNS Foundation’s Founder, Fia Richmond.

Recap:  In January, scientists from both academia and the private sector sat down with the Pediatric Brain Foundation team and advocacy leaders at our 2013 Scientific Workshop: The Best Next Steps to Advance the Field of Pediatric Neurology.  Through an ambitious agenda, we identified barriers to progress and discussed innovative and practical ways to navigate these hurdles.  With support from the California Institute of Regenerative Medicine (CIRM), representatives from Harvard Medical School, Sanford Burnham Medical Research Institute, University of California, Texas Children’s Hospital and other institutions offered expert analyses and opinions.

The workshop took place at a crucial time, as the NIH—the largest funder of science on the planet—continues to operate with an atrophied budget.  The agency is receiving unprecedented numbers of funding requests and the overwhelming majority are being denied; labs and research facilities across the United States are closing and we are in danger of experiencing a “brain drain,” as scientists are lured onto alternative paths, sometimes leaving the field entirely, to pursue their work. 

In the 1990s, President Clinton increased the NIH budget and both the academic and private sector teemed with scientific progress.  Workshop participants agreed that an increase in the NIH budget would provide an immense boost to field of pediatric neurology and science as a whole.  While this may seem obvious, it is important to reinforce and remain steadfast in pressuring policy makers to protect scientific research.

One idea, which has come up time and again in past workshops, is the need for a Natural History Database of Pediatric Neurological Diseases.  A database that includes the complete medical histories of children with common, rare and undiagnosed neurological conditions would allow researchers to search for commonalities among patients and amongst very complex disorders.  This would be especially useful when studying rare or undiagnosed diseases, as children afflicted are often too sick and fragile to travel across the country to different laboratories and clinics.  While many similar databases exist for specific diseases, there is no single collection of pediatric neurological conditions.

Pediatric Brain Foundation has a long history of bringing together scientists, advocates, parents, policy makers and representatives from the private sector.  Pediatric Brain Foundation is uniquely suited to champion a project like a Natural History Database.  While we are still flushing out the details and researching the best strategies to bring this idea to fruition, we are confident Pediatric Brain Foundationcan act like a force multiplier and rally the field to complete this project. 

Despite the dearth of scientific funding, our country is full of great minds working on great challenges, and progress is occurring everyday.  The question for Pediatric Brain Foundation is: What can we do today to lay the groundwork for breakthroughs tomorrow?