Date: July 11th, 2002
Subject: Pediatric Brain Foundation’s (Formerly CNS Foundation) 2nd Annual Scientific Advisory Board Think Tank
Location: Boston, MA
Attendees: Pediatric Brain Foundation Board members, parent advocates, affected children, and scientists including Harley Kornblum, Evan Snyder, Ron McKay, Mark Noble, Mahendra Rao, and Jeffrey Macklis.
Recap: Integral to Pediatric Brain Foundation’ s mission is the collaborative relationship between the parents and the scientists. The key role that the parents play is to present to the scientists the critical data that they have acquired by observing their children’s behavior. The goal is not only to highlight the sense of urgency we parents feel on a daily basis, but to also help determine goals and strategies for future research.
A Parent’s Perspective on the Pediatric Brain Foundation’s 2nd Annual Scientific Advisory Board Think Tank
By Sue Ellen Strong,
It was with great anticipation that I attended the Pediatric Brain Foundation’s 2nd Annual Scientific Advisory Board Think Tank.
I was attending not only as a board member of Pediatric Brain Foundation, but also as mother to Katherine, age five, who was there with me to meet these prestigious scientists. The parents and their children were invited to actively participate in this Think Tank. Integral to Pediatric Brain Foundation’s mission is the collaborative relationship between the parents and the scientists. The key role that the parents play is to present to the scientists the critical data that they have acquired by observing their children’s behavior. The goal is not only to highlight the sense of urgency we parents feel on a daily basis, but to also help determine goals and strategies for future research.
The scientists embraced our stories with heartfelt concern. All of the children presented are undiagnosed, medical mysteries. The Scientific Advisory Board (SAB) members listened and asked questions as parents told of experiences with traditional medicine, as well as many alternative therapies. They acknowledged the level of desperation expressed by all of the parents in the search for any treatment that may benefit their children. The scientists present were clear in their concerns. As Harley Kornblum, M.D., Ph.D., Associate Professor of Molecular and Medical Pharmacology and Pediatrics at UCLA School of Medicine, said in addressing the parents, “We, as the medical community, have failed you and your children.” Parents of special needs children have felt this for a very long time. And even though we know this to be true, it was very affirming to have a representative of the medical establishment validate it. It was abundantly clear that the scientists who joined with us want to improve upon this situation. The presence of these scientists around the table was a testament to this truth and to their desire to change this reality for us in the future.
SPOTLIGHT ON THE CHILDREN
The scientists were particularly interested in any family history of brain disorders, the point at which parents noticed changes, and positive responses to any form of treatment. When all of the mothers noted that at birth they were told they had healthy children, Evan Snyder, M.D. Ph.D. Assistant Professor of Neurology at Harvard Medical School, explained that testing for brain dysfunction at early stages of development is very primitive. Part of the reason for this, he noted, is that a baby needs only limited brain development to perform basic body functions for the first several months. The discussion of the issues facing the children present led the group to address the need for research models that accurately represent pediatric neurological disorders. “Children with neurological problems are under-represented (in the research community),” said Evan Snyder. He stated that “there are many foundations for specific diseases, and Pediatric Brain Foundation can help by getting to the root causes of broad neurological problems.” Ron McKay, Ph.D., Chief of the Laboratory of Molecular Biology at the National Institute of Neurological Disorders and Stroke (NINDS), highlighted the need to know how the brain develops before it can be repaired.
This collaborative forum raised many fundamental issues that will need to be addressed in order to move forward the research required to help our children, including identifying the critical things to look for in the developing brain to assess normal development vs. genetic defects and damage. “Children with neurologically based problems present with hundreds of different conditions,” said Mark Noble, Ph.D., Professor of Genetics at the University of Rochester School of Medicine. “Are there general principles we can focus on that apply to a broad range of neurological problems,” he asked, “or is it the case that every individual syndrome, or every individual child, will require a customized approach?”
TOOLS OF THE TRADE
One of the most encouraging aspects of this meeting was that these scientists have demonstrated in their laboratories that brain repair is possible in animal models. They now need to understand the repair mechanisms on a cellular level. This will allow them to develop therapeutics that will result in functional changes for our children. Repair is possible, and the question now is how to move most effectively to enable such repair to occur.
Mahendra Rao, M.D., Ph.D., Chief Investigator of the Developmental Neurobiology Section of The National Institutes of Aging, initiated a discussion regarding the need for appropriate diagnostic tools to characterize cells and measure changes in cellular metabolism, especially for animal models that will effectively represent the developing human brain. These animal models will be crucial in translating the basic science into clinical applications.
Jeff Macklis, M.D., D.HST, Associate Professor of Neurology at Harvard Medical School, explained how understanding the molecular pathways will allow the brain cells to be manipulated in such a way as to renew their plasticity, i.e. their ability to repair themselves. He added that advances in imaging technology will also allow us to better understand brain abnormalities and subtle cellular changes.
Though it may seem that there were many more questions posed than answers offered, we must remember that in order to find the right answers, we must ask the right questions. The Think Tank effort was an important start in helping us to focus on these questions, and to build the coalitions with leading scientific laboratories that will enable these questions to be answered.
I left the meeting feeling comfort in the fact that there are leading scientists who believe that brain repair and regeneration are possible, and that they feel a responsibility to help focus research efforts on therapies for our children. The Pediatric Brain Foundation was conceived as a parent-scientist collaboration. This meeting was a perfect example of how parental involvement is critical to focusing the research efforts and driving the urgency of therapeutic solutions home to those who have the ability to find the answers. Pediatric Brain Foundation can help expedite this research by filling in gaps of knowledge that are roadblocks to progress, by funding projects which answer fundamental questions, by challenging scientists to pursue the fastest path to clinical applications, and by encouraging young scientists to embark on a career in this field. They are listening!
Do what you can to invest in one or more of these efforts. We look forward to sharing progress reports with you soon! Here’s to a brighter future for our kids!