Subject:  Neurotrophic Factors as Therapeutic Treatments for Pediatric Neurological Disorders: Moving the Field Forward

Location:  San Jose, California

Chair:  Dr. William Mobley, MD, PhD; Professor and Chair, Department of Neurosciences, University of California, San Diego School of Medicine

Attendees:  A-T Children’s Project representatives, Pediatric Brain Foundation representatives including Dr. Evan Snyder, Dr. Mark Noble, Dr. Harley Kornblum, Dr. Theo Palmer, and Dr. Clive Svendsen

Recap:  On February 28 through March 3rd, 2006, Pediatric Brain Foundation co-hosted a scientific workshop in San Jose, California, entitled Neurotrophic Factors as Therapeutic Treatments for Pediatric Neurological Disorders: Moving the Field Forward.  This meeting, held in collaboration with the A-T Children’s Project, focused some of the top researchers in the field of neurotrophin biology on new treatment strategies for special-needs children.  Neurotrophins (pronounced neu-ro-tro-fins) are protein factors within the brain that promote the assemblage of brain cells into functional networks.

Under the guidance of Workshop Chair Dr. William Mobley, Pediatric Brain Foundation and ATCP charged meeting participants with the task of re-evaluating neurotrophins in the context of childhood brain disorders.  The meeting began in earnest on March 1 with a day-long review of new findings in neurotrophin research and an overview of how these molecules play into developmental disorders of the central nervous system.  Pediatric Brain Foundation Scientific Advisory Board members Evan Snyder, Mark Noble, Harley Kornblum and Theo Palmer were among those leading these discussions.

The following day imposed a clinical focus upon the Workshop.  We began with a look at previous successes and failures in the clinical application of neurotrophins, an appraisal in which  Scientific Advisory Board member Clive Svendsen played a role.  This section was then followed by a lively exchange on potential uses of these molecules as a treatment that could be ready for initial clinical evaluation in three years or less.  The Workshop concluded its second full day with attendees seeking guidance about possible pre-clinical requirements and clinical trial methods from representatives of the Food and Drug Administration.

By the Workshop’s final day, attendees looked for a disease where the direct administration of a neurotrophin might accomplish short- or long-term brain repair, ultimately focusing on the delivery of a molecule called IGF-1 to children with ataxia-telangiectasia (abbreviated A-T). 

Significantly, Workshop participants agreed to contribute to an on-going effort to achieve this goal.  Pediatric Brain Foundation is now working with the ATCP and a team of Workshop attendees to develop preliminary patient inclusion criteria as a first step toward the proposed IGF-1 clinical trial.  While there is tremendous enthusiasm amongst this group for the project, much work needs to be done – and many critical regulatory hurdles cleared – before the promise of the Neurotrophic Factors workshop is fully realized.