The Autonomic Disorders Consortium (ADC) was established as a Rare Disorders Clinical Research Consortium (RDCRC) in August 2009. The team includes physicians, scientists, nurses, patients and support groups dedicated to finding new therapies to treat and cure these diseases. Diseases the ADC plans to focus on include:
- Multiple System Atrophy (MSA)
- Pure Autonomic Failure (PAF)
- Parkinsonism with Autonomic Failure
- Lewy Body Disease
- Autoimmune Autonomic Ganglionopathy (AAG)
- Postural Tachycardia Syndrome (POTS)
- Dopamine β-Hydroxylase Deficiency
- Norepinephrine Transporter Dysfunction
- Familial Dysautonomia
- Baroreflex Failure
- Neurally Mediated Syncope
- Takotsubo Syndrome
The Autonomic Disorders Consortium's home institution is Vanderbilt University. The Consortium consists of five sites:
- Vanderbilt University
- New York University
- Beth Israel Deaconess / Harvard
- Mayo Clinic
- NINDS / NIH
The mission of the ADC is to study autonomic disorders to develop novel therapies aimed not only at improving quality of life, but also altering the course of the disease. The ADC is multidisciplinary and draws strong support from our patients and their support organizations. The Consortium joins with patient support groups to harness the knowledge and energies of physicians and investigators in the major centers where these patients are cared for, so that they can discover ways to treat and to cure these diseases.
Goals for the Autonomic Disorders Consortium
We plan to meet our goals through:
- Natural history studies
- Therapeutic trials
- Novel interventions which include:
- A potentially disease arresting intervention in MSA.
- Immunomodulatory therapy
- Natural History of Autonomic Failure
- A bionic baroreflex
- A dopa-decarboxylase antagonist
- Patient registries
- Data / Specimen banks
- Training physicians and scientists in the investigation and treatment of rare autonomic disorders
- Standardizing evaluation of autonomic function
- Learning about the long-term prognosis of the disorders
Autonomic disorders cause loss of regulation of the heart, blood vessels, stomach, bowel and bladder. Affected patients may have palpitations or lose consciousness, and some have a rapidly fatal course. The Autonomic Disorders Consortium joins with patient support groups to harness the knowledge and energies of physicians and investigators in the major centers where these patients are cared for, so that they can discover ways to treat and to cure these diseases. The greater the collaboration between doctors and patients, the more we can learn about these disorders. This important first step is necessary if we are ever to find genuinely effective and curative treatments.
The Vanderbilt Autonomic Dysfunction Center was established in 1978 as the first international center for patient care, research, and training focusing exclusively on disorders of the autonomic nervous system. Its creation brought together in one site a cadre of physicians, scientists and nurses who could foster a balanced scientific approach to elucidation of the etiology and optimal therapy of these disorders. Over the years, many people have contributed to this effort at Vanderbilt. Many of the young scientists and physicians who have trained here have gone out and established similar centers in other parts of the world. Others have taken positions at academic research centers and pharmaceutical firms where they are seeking improved drugs to treat these disorders.
Investigators in Vanderbilt's Autonomic Dysfunction Center have identified previously unrecognized disorders, including dopamine-beta-hydroxylase deficiency, a syndrome in which patients have a congenital absence of norepinephrine and epinephrine, and norepinephrine transporter deficiency, a disorder in which there is impaired clearance of norepinephrine from the synaptic cleft. The investigators have introduced novel therapeutic modalities for the management of orthostatic hypotensive patients. They also study baroreflex failure in human subjects. Orthostatic intolerance, nitric oxide mechanisms in blood pressure regulation, and the dysautonomia of hypoglycemia are additional current research topics.
All consortium sites (Mayo, Harvard, New York University, the National Institutes of Health and Vanderbilt) have long traditions in discovery and treatment of autonomic disorders.