Date:  July 15-16, 2010

Subject:  Accelerating Therapeutic Development for Pediatric Neurological Disorders: Establishing Clinical Centers of Excellence

Location:  Washington, D.C.


  • Brad Margus, CEO, Envoy Therapeutics and Volunteer President, and Co-Founder, A-T Children’s Project
  • Dr. William Mobley, Chair, Department of Neurosciences, School of Medicine, University of California at San Diego 
  • Dr. Evan Snyder, Program Director, Professor Stem Cells and Regenerative Biology, Sanford-Burnham Medical Research Institute

Attendees:  Pediatric neurologists, scientists, pharmaceutical executives, venture capitalists, leaders of disease-specific nonprofit organizations as well as representatives of the Food and Drug Administration and the National Institute of Neurological Disorders and Stroke.

Recap:  On July 15 & 16, 2010, Pediatric Brain Foundation hosted a symposium in Washington D.C. that brought together policy makers, researchers, non-profit organizations, and industry to address the barriers to clinical trials and treatments for children affected by neurological conditions.  The meeting, Accelerating Therapeutic Development for Pediatric Neurological Disorders: Establishing Clinical Centers of Excellence, had a primary goal of fully fleshing out the feasibility and details of creating a network of locations throughout the United States that would ultimately be able to coordinate, plan and execute pediatric brain studies and then share the results to an alliance of physicians, scientists, health care providers, federal agencies, industry and patient advocates.  

Event co-chairs, Brad Margus (CEO, Envoy Therapeutics and Volunteer President and Co-Founder, A-T Children’s Project), Dr. William Mobley (Chair, Department of Neurosciences at the University of California, San Diego School of Medicine) and Dr. Evan Snyder (Program Director, Professor Stem Cells and Regenerative Biology, Sanford/Burnham Institute), facilitated the discussions and provided focus and direction throughout the two days. 

The list of attendees was stellar. Thirty people representing various stakeholders attended and contributed a diversity of points of view.  Top staff members from NINDS and from FDA participated, as well prominent researchers and pediatric neurologists.  Several Executive Directors and key staff from non-profit groups were there, including:  Citizens United for Research in Epilepsy (CURE), R.A.R.E. Project, National Tay Sachs & Allied Diseases, A-T Children’s Project, Faster Cures/The Center for Accelerating Medical Solutions and National Down Syndrome Society.   In addition, corporate executives from Pfizer, Athersys, and StemCells Inc. and venture capitalists rounded out the group.  

“We went from the Valley of Death, to a Mountain of Hope.” -Dr. William Mobley

CNS Foundation is working on the extensive follow-up and coordinating the efforts of the Co-Chairs in getting the Centers of Excellence for Pediatric Neurological Disorders off the ground.  Although the details are still being hammered out, it is proposed that the network be organized and managed under several Centers of Excellence.  A hub and spokes model would establish a highly organized trials network populated by academic Child Neurologists across the United States.  

The network would also be charged with:

  • Providing training for Child Neurologists and their physician and nurse colleagues in clinical trial design management, and interpretation
  • Exploiting existing infrastructure for clinical trials design, execution, and, evaluation
  • Where needed, creating such an infrastructure
  • Evaluating emerging basic science discoveries as potential treatments
  • With colleagues at the FDA and industry, assessing the feasibility of developing discoveries into drugs or other therapeutic modalities
  • Enlisting international partnership as necessary to advance the mission
  • Communicating the results of studies to colleagues and to patients and their advocates, including assembling a searchable accessible database