Over the last few years, the Children’s Neurobiological Solutions (CNS) Foundation and the A-T Children’s Project (A-TCP) began to realize that a critical gap existed in the path to bringing potential therapies to the clinic for children with neurological di sorders. An organized network of multidisciplinary, well-funded clinicians experienced in performing clinical trials for rare, pediatric neurological disorders, like ataxia-telangiectasia (A-T), did not exist.
Because the absence of such a network would significantly slow the rate at which new therapies could be tested in children with brain diseases, the A-TCP and CNS Foundation set out to find a way to fill this gap.
Recently, the two foundations organized a workshop near Washington, D.C. to address the need for a network of experts in pediatric neurological trials. In attendance were rare disease advocates, neonatal and pediatric neurologists, venture capitalists, pharmaceutical company representatives and representatives from the FDA and National Institutes of Health (NIH). Representing the National Institute for Neurological Disorders and Stroke (NINDS) at the NIH was Petra Kaufmann, MD, an expert clinical trialist for neuromuscular disorders who had recently joined the NINDS as Director of the Office of Clinical Research. Dr. Kaufmann immediately saw how the need to fill this gap in bringing therapies to children paralleled an NINDS initiative to establish a Network for Excellence in Neuroscience Clinical Trials, given the acronym NEXT.
As a result of this interaction, within months of the workshop, the NINDS released a “Request for Applications” for NEXT that included support for clinical research sites capable of performing trials for children with rare neurological diseases. The CNS Foundation and A-T Children’s Project thank Dr. Kaufmann and the NINDS/NIH not only for recognizing this important bench-tobedside gap involving children, but also for filling it with unprecedented speed.