Dysautonomia Foundation, Inc.
| FD Research at
NYU Dysautonomia Center |
The Dysautonomia Foundation encourages and financially supports clinical, genetic and basic science research into FD by funding the work of eminent scientists throughout the world. In 2007, the foundation constructed the world's first autonomic disorders clinical research laboratory with a primary focus on FD. It is headed up by Horacio Kaufmann, MD. Dr. Kaufmann, one of the world's foremost authorities on autonomic disorders, holds an endowed professorship in FD research at NYU School of Medicine and has appointments in the departments of Neurology, Medicine and Pediatrics. |
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THE DYSAUTONOMIA CENTER AT NYU SCHOOL OF MEDICINE The Dysautonomia Research Laboratory has a number of research projects underway at the NYU Dysautonomia Center. Below is a list of the current research projects, including a brief summary of funding, objective, approval and eligibility for each project.
Rare Autonomic Disorders Consortium NYU’s Dysautonomia Center was awarded 5 years of funding from the National Institutes of Health (NIH) together with Vanderbilt, Harvard, Mayo Clinic and Intramural NIH to form a Consortium of leading investigators to study rare autonomic disorders. The overall objective of this project is to define the natural history of rare autonomic disorders and develop novel treatment plans aimed at improving quality of life and altering disease prognosis. Carbidopa for the treatment of nausea and vomiting in FD The National Institutes for Health (NIH) awarded Dr. Kaufmann and his team funding from the Food and Drug Adminstration (FDA) to conduct a clinical trial of carbidopa for the treatment of nausea in FD. Carbidopa has been used since the 1970’s as an adjunct therapy for patients with Parkinson’s disease to prevent vomiting. Preliminary data from the Dysautonomia Laboratory suggests that the drug may have the same effect in patients with FD. This study is approved by NYU’s Institutional review board (H#09-0011). Dr. Kaufmann holds an IND from the Food and Drug administration for the use of carbidopa in patients with FD. The study is open to patients with FD over the age of 12 who suffer from nausea and vomiting. Parents and/or patients should contact the study coordinator Dr. Lucy Norcliffe-Kaufmann at 212-263-7225 for more details. The pathophysiology and natural history of FD A major research project of the Dysautonomia Center is to explain the autonomic abnormalities in FD. The project also focuses on studying the natural history of FD and other HSANs and assessing which therapeutic interventions improve quality of life and survival. This study, funded by the Dysautonomia Foundation, is approved by NYU’s Institutional review board (H#07-938) and is open to all patients with FD. Domperidone for the treatment of gastrointestinal dysfunction in autonomic disorders Domperidone is widely used throughout Europe and Canada for the treatment of gastrointestinal motility disorders (such as delayed gastric emptying and nausea). Dr. Kaufmann was given an Investigational New Drug (IND) approval from the U.S. Food and Drug Administration to use domperidone for the treatment of gastrointestinal problems in patients with autonomic disorders and has approval from NYU’s Institutional Review Board to use this drug (H#09-0175). Droxidopa for the treatment of orthostatic hypotension Dr. Kaufmann is the Principal Investigator of a multi-center international phase III clinical trial of droxidopa for the treatment of orthostatic hypotension in patients with autonomic failure. Droxidopa is a synthetic amino acid that when taken by mouth is converted into norepinephrine. The drug is currently approved and widely used in Japan for the treatment of orthostatic hypotension. These studies are currently open to patients with autonomic failure. These trials are approved by NYU’s Institutional review board (H#07-950 and #08-224). Nerve function in FD In collaboration with Dr. Joel Gutierrez, Professor of Neurology and Clinical Neurophysiology from Havana University School of Medicine in Cuba, the Dysautonomia Research Laboratory is currently conducting a study of nerve function in patients with FD and carriers of the FD gene mutation. This study, funded by the Dysautonomia Foundation, is approved by NYU’s Institutional review board (H#07-938) and is open to all patients with FD and carriers of the gene mutation. Kinetin in FD Kinetin is a nutritional supplement that has been shown to correct the gene splicing defect and protein deficiency in human FD cell lines and in transgenic mice models. Current studies are underway at the Dysautonomia Center to determine whether kinetin corrects genetic misreading and enzyme deficiency in FD patients. This study is funded by the Dysautonomia Foundation and is approved by NYU’s Institutional review board (H#09-0762). The study is open to patients with FD over the age of 18.
THE DYSAUTONOMIA CENTER AT HADASSAH
MEDICAL SCHOOL
Dr. Channa Maayan is the principal investigator at the Israeli
Dysautonomia Center and maintains close collaborations with
appropriate specialists in many other areas. Dr. Maayan is a
pediatric pulmonologist. He work focuses on describing the
functional problems of patients with familial dysautonomia. She
is the Principal Investigator for several FD clinical research
projects and is a collaborator on many of the FD basic science research
protocols currently underway in Israel.
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