Multiple System Atrophy Research Program
The Division of Autonomic Disorders, in conjunction with NYU Langone’s Dysautonomia Center, leads a comprehensive clinical research program dedicated to multiple system atrophy (MSA). As a world-class reference center for MSA, the Multiple System Atrophy Research Program provides direct access to clinical care and support to more than 100 people living with MSA.
We offer patients the opportunity to participate in research studies focused on understanding the causes and evolution of MSA; developing and approving new symptomatic treatments of MSA; and discovering treatments that can stop or slow the progression of neurodegeneration in MSA.
Our goal is to improve the quality of life of people who have MSA and their families, and eventually find a cure. A distinctive hallmark of our program is its translational approach to research. Patients can participate in cutting-edge research studies at various stages of the disease while also receiving the best clinical care from our dedicated team, led by Horacio Kaufmann, MD.
Global Multiple System Atrophy Patient Registry
In collaboration with the Rare Diseases Clinical Research Network, we maintain the Global MSA Patient Registry (GLoMSAR), which connects people who have MSA and their caregivers to physicians and researchers focused on the disease. More than 1,000 people with MSA are part of the registry, which also provides them with updates on treatments, news, and clinical trial opportunities. The registry also enables neurologists who specialize in treating MSA to contact eligible patients to participate in clinical trials and speed up the testing of potential new therapies.
We administer surveys to GLoMSAR participants to better understand specific features of MSA and capture each patient’s perspective of the challenges of living with MSA. Participate in a GLoMSAR survey.
Preparing for Clinical Trials
NYU Langone’s Dysautonomia Center is a founding member of the Autonomic Disorders Consortium, a National Institutes of Health (NIH)–funded initiative to bring new therapies to patients with rare autonomic disorders. Our MSA Research Program is the main site of an international collaborative project to define and understand the natural history of MSA, to map how and when the disease begins, and how it evolves over time. Data collected in this study includes information about the disease onset of each patient, serial neurological examinations, neuroimaging, blood samples, and genetic analysis. This information is key in planning clinical trials that test new therapies for MSA.
The MSA Research Program is dedicated to developing tools to improve the diagnostic accuracy of MSA, particularly in the early stages. We are researching methods to help clinicians identify people with MSA, before the characteristic abnormalities emerge so that there may be a chance to prevent them. Together with other members of the Autonomic Disorders Consortium and the Movement Disorders Society, we are spearheading an update of the diagnostic criteria for MSA. Dr. Kaufmann leads this task force to update the MSA diagnostic criteria.
We are also collaborating with basic researchers to discover new biomarkers that can accelerate the diagnosis of MSA and distinguish it from other similar disorders, such as Parkinson’s disease.
Developing New Treatments for MSA and Its Symptoms
NYU Langone’s Dysautonomia Center has a long history of developing and helping approve drugs for the symptomatic treatment of MSA. In the last 10 years, researchers at our center have led the development and approval of midodrine and droxidopa (Northera), two drugs now used for the treatment of orthostatic hypotension in people who have MSA.
Having been involved in pivotal trials for drug approval, we understand the care and attention to detail that is needed to test new treatments to find ones that are safe and effective for people with MSA. We review the best evidence to decide which potential treatments hold the most promise as emerging therapies for MSA. We also play an active role in designing research protocols. Our nursing and medical staff are trained to monitor patient safety when participating in research trials, and we ensure that all our studies meet regulatory requirements necessary for good clinical practice.
Dr. Kaufmann and Jose-Alberto Palma, MD, PhD, are the lead investigators for the NIH–National Institute of Neurological Disorders and Stroke (NINDS) sponsored study Clinical Trial Readiness in Multiple System Atrophy. The goal is to develop an outcome assessment that can be used in treatment trials to accurately measure disease progression and detect if a new medication is effective in slowing down the progression of MSA.
Clinical Trial Readiness in Multiple System Atrophy is an international, multicenter, prospective, observational study enrolling patients with multiple system atrophy and following them for 12 months. The study will leverage the existing infrastructure for the Autonomic Rare Disorders Clinical Research Consortium (RDCRN), the European MSA Study Group, and the major MSA patient advocacy groups.
With a grant from the NIH, we tested sirolimus, a medication used to prevent organ transplant rejection, to slow the progression of MSA by enhancing the removal of toxic proteins at the cellular level. Although this study was ended early due to interim analysis showing that the medication was not effective in slowing the progression of MSA, it provided important information about the progression of MSA which can help guide the design of future clinical trials.
We continue to have a number of clinical trial opportunities that are open to patients with MSA according to their specific needs.
We are currently testing ampreloxetine, a new drug for orthostatic hypotension specifically designed for people with MSA.
We are also conducting studies to test possible disease modifying treatments by using antibodies to remove abnormal alpha-synuclein, a protein thought to mediate disease progression in MSA and Parkinson’s disease. This may be done by injecting the antibodies (TAK-341 and AMULET studies for MSA), or by using a vaccine to help the body produce these antibodies (UB-312 for MSA and Parkinson’s disease).
Rare diseases research requires a collaborative approach. NYU Langone’s Dysautonomia Center is the central site of the largest-ever natural history study of MSA, coordinating more than 20 recruiting sites around the world. We implemented this research so the data can be safely shared with collaborators to learn more about the disease. Our collaborative teams have access to MSA data so that they can answer pressing research questions in a large cohort of closely followed patients around the world.
We also share biospecimens with our research collaborators to speed up the development of biomarkers that can be used for clinical trials or diagnosis. Currently, we are collaborating on research studies to understand the genome, blood-borne biomarkers of disease, and neuroimaging changes.
The Dysautonomia Center also organized the 6th International MSA Congress, held in March 2018 in New York City. This was the biggest ever event devoted to MSA, gathering together international experts on MSA, as well as patients. This forum fostered collaboration and helped to define a roadmap for MSA initiatives.
Educating Future MSA Specialists
The Division of Autonomic Disorders offers a United Council for Neurologic Subspecialties (UCNS)–accredited Autonomic Disorders Fellowship. Young physicians entering the program learn to diagnose and manage patients with autonomic disorders, with special emphasis on MSA. During the program, fellows are introduced to clinical research methodology and present their results at international conferences and in renowned publications. This program is designed to prepare the future leading experts on MSA research.
For more information about the MSA Research Program, please contact Lee-Ann Lugg, program coordinator, at email@example.com or 212-263-7225.