A nationwide research group headed by Vanderbilt University’s David Robertson, M.D., has received another round of funding from the federal government to continue studies of rare neurodegenerative diseases and disorders affecting blood pressure.
The Autonomic Rare Diseases Clinical Research Consortium is one of 22 Rare Disease Clinical Research Consortia to receive funding this year from the National Institutes of Health (NIH).
The consortia bring together physicians and scientists working collaboratively to find the fundamental causes of these diseases in order to develop effective therapy. Many are caused by rare genetic mutations but others may have environmental causes.
“The importance of this NIH program is that it enables us to study patients whose diseases are extremely rare,” said Robertson, the Elton Yates Professor of Medicine, Pharmacology and Neurology and director of Vanderbilt’s Clinical Research Center.
“Only through such collaboration could we hope to understand the cause of their disease, and hopefully find a way to make their lives better,” he said.
The Autonomic Consortium includes four centers in addition to Vanderbilt: Beth Israel/Harvard University, Mayo Clinic/Rochester, New York University and the intramural NIH. Major support groups for autonomic diseases such as the MSACoalition and Dysautonomia International are engaged and participate at many levels in the activities of the consortium.
Over the years the Autonomic Consortium has received $6.4 million in NIH grants, including $562,000 in the current fiscal year.
The consortium focuses particularly on the neurodegenerative diseases such as the Synucleinopathies: multiple system atrophy (MSA), Lewy body dementia (LBD), and pure autonomic failure (PAF). Research by consortium members has led to the publication of 129 scientific papers and two textbooks.
The Vanderbilt Autonomic Dysfunction Center also studies the pathophysiology and therapy of postural tachycardia syndrome (POTS), baroreflex failure, chronic fatigue syndrome, and neurally mediated syncope, and includes a robust component that educates future clinical investigators.
Key Vanderbilt investigators include Italo Biaggioni, M.D., Satish Raj, M.D., Andre Diedrich, M.D., Ph.D., Emily Garland, Ph.D., Cyndya Shibao, M.D., Alfredo Gamboa, M.D., Amanda Peltier, M.D., and Luis Okamoto, M.D.
Earlier this year researchers at Vanderbilt and the University of Oklahoma reported that circulating “autoantibodies” possibly triggered by a viral illness may contribute to some patients with POTS, an abnormally rapid heart rate (tachycardia) upon standing that affects 500,000 Americans, mostly young women.
