Recognizing both medical progress and unmet needs in the field of developmental disabilities, The Children's Hospital of Philadelphia will house the Network Coordinating Center for a newly established collaborative organization, the Developmental-Behavioral Pediatrics Research Network.
Launched this year with a three-year, $200,000 per year grant from the federal government's Maternal Child Health Bureau, the Network brings together experts from 12 leading pediatric programs to focus on a broad range of neurodevelopmental disabilities, notably autism spectrum disorders, attention-deficit hyperactivity disorder (ADHD), and intellectual disability (formerly referred to as mental retardation, and affecting an estimated 2 to 3 percent of children).
These disabilities often involve problems in learning, attention, inappropriate behaviors, sleep and social skills.
"These conditions are highly challenging to families, educational systems and caregivers," said Nathan J. Blum, M.D., chair of the Network's Executive Committee, and the director of the Leadership Education in Neurodevelopmental Disabilities (LEND) Program at The Children's Hospital of Philadelphia. "Two of our largest challenges are how to best individualize treatments to our patients, and how to develop more effective interventions for these conditions."
Even for the most effective treatments, added Blum, such as using stimulant medication for ADHD, a significant number of patients do not respond well to existing treatments. For many other developmental disorders, treatments remain to be developed or adequately evaluated.
By fostering collaboration among leading clinical and research programs, said Blum, the Network will be able to sponsor multi-site studies in many patients to provide a large enough sample size to properly evaluate new treatments as they are developed.
The Network will also provide opportunities to make use of recent advances in genetics and neuroimaging, as applied to neurodevelopmental disorders. As those technologies may lead to better diagnostic tools, they offer the potential of earlier diagnosis and possibly earlier interventions. In particular, genetic studies such as those conducted at Children's Hospital's Center for Applied Genomics and other facilities are identifying biochemical pathways that are altered in neurodevelopmental conditions.
"We expect that greater knowledge of these biological pathways will allow scientists to develop drugs to target specific disabilities and potentially reverse learning deficits," said Blum. "Having a developmental-pediatrics research network will provide greater opportunities to investigate the effectiveness of new drug candidates, and more rapidly translate basic science findings into eventual clinical treatments."
In its initial stages, said Blum, the new Network will develop the infrastructure to build itself, will agree on a research agenda, will standardize data collection from its member sites, and will initiate smaller projects to share data. The Network Coordinating Center, based at The Children's Hospital of Philadelphia, will organize the Network's central administrative functions. The Network will then select and implement new research studies.
Source: The Children's Hospital of Philadelphia