Multiple system atrophy (MSA) is a relentlessly progressive parkinsonian disorder that affects an estimated 15,000 Americans (1). There are no available disease-modifying treatments (2). MSA is characterized by difficulties with movement, which are poorly responsive to all known anti-parkinsonian medications (1, 3, 4). Another characteristic feature of the disease is failure of the autonomic nervous system with progressive loss of control of the heart, blood vessels, gut and bladder. Age of onset is typically in the mid-50s (5) and average survival is 8-years (1, 3, 4). It affects both men and women and has no clear ethnic or racial susceptibilities (6). Its rarity requires a collaborative approach to power clinical trials and develop better treatments (7). The 6th International MSA Congress is a 2 and a half-day organized meeting focused on MSA, from the molecular mechanisms to the bedside. The first meeting was held 1997 with subsequent meetings in 2004, 2007, 2012 and 2016. These have lead to a number of productive basic science and clinical collaborations (4, 5, 8-11). The 2018 Congress take place in New York City on March 1st to 3rd and will bring together a diverse expert faculty chosen for their accomplishments in MSA within the last 2-years. Day 1 will be devoted to the neurobiology, biomarkers and disease-modifying treatments. Day 2 will focus on early diagnosis, atypical presentations, and symptomatic treatment. The conference will have a plenary lecture, short comprehensive updates, round table discussions, breakout sessions, and activities for young investigators include a poster session. The Autonomic Disorders Consortium was founded in 2009 as part of the NIH Rare Disease Clinical Research Network (RDCRN). The Consortium's flagship project is the Natural History Study of MSA and lead by investigators at NYU School of Medicine (Project 1: U54NS065736). This project is now the largest natural history study of MSA in the world. This R13 grant application proposes to support young faculty members to attend the Congress. Training sessions will address key clinical questions including: 1) How to identify MSA patients in the pre-motor stage? 2) How to systematically follow the progression of a patient? and 3) Research strategies for clinical trials. Members of the advocacy community, industry, and the RDCRN Steering Committee will also participate in the Congress. The sessions will be video documented, published in biomedical journals, and transmitted to the patient community online. The meeting will be planned with the NYU School of Medicine Continuing Medical Education (CME) Office. Bringing together leaders in MSA research with advocacy groups and industry representatives in an intensive program will further solidify our global efforts to collaborate and find new treatments for this brutal disorder.
The 6th International Multiple System Atrophy (MSA) Congress will bring together a diverse faculty to focus on recent developments in the pathophysiology and treatment of the rare neurodegenerative disease MSA. Funds are requested in this R13 application to bring junior faculty to the Congress to learn about the pathogenic role of the protein alpha-synuclein, its prion-like behavior, the current status of clinical trials, and hear expert opinions on managing the symptoms of MSA. The Congress will solidify collaborations and provide a framework for a collaborative path forward in this rare disease.
