The National Institutes of Health (NIH) began an initiative to create collaborative consortia to bring together investigators working on rare (orphan) diseases. In 2009 five academic medical centers in the United States joined to create the Autonomic Disorders Consortium (ADC). NYU’s Dysautonomia Center is the site leading the natural history study. The project documents the clinical features of patients with rare autonomic diseases overtime. Amidst increasing competition, NIH has renewed funding for the Consortium. The grant award to was for four years.
Why study the natural history of a disease?
Large and well thought out natural history studies provide the groundwork necessary for planning clinical trials to test new treatments. They help us decide what endpoints to measure in a trial and how they should be measured. They provide the means to understand how much patients vary from one another and allow us to reliably estimate the number of patients needed for a successful clinical trial. They also help us see which therapies we use in the clinic prolong survival and improve quality of life.
What the plans for the future?
Our national network of collaborators gave us strength in numbers. Now, the natural history study is expanding into a global international project, with the Dysautonomia Center leading the effort. We are also concentrating our efforts on defining that natural history of multiple system atrophy, a rare and fatal autonomic disease, still with a lot of unknowns. We are now searching to find ways to spot neurological diseases in the pre-motor stages, before they spread to the brain. Our hope is that we can intervene sooner and change the prognosis for these patients.
A united approach
Partnership between government funding agencies and patient advocacy groups has been essential in our success. The MSA Coalition has pledged support to reimburse new sites and help towards their costs of collaborating data. The Dysautonomia Foundation’s unwavering support of our mission makes this entire endeavor possible. The Rare Disease Clinical Research Network (RDCRN) at the NIH continues to support our mission to advance our understanding of rare autonomic disorders and improve the lives of patients.
Join the Rare Disease Clinical Research Network Registry and stay up to data with the Autonomic Disorder’s Consortium.