Nine research projects will be awarded funds to advance global efforts to achieve a greater understanding of multiple system atrophy, a rare and devastating neurodegenerative disease. This year’s awards represent a true international collaboration among MSA charitable organizations interested in funding the most promising, scientifically vetted research proposals. The MSA Coalition is especially pleased to welcome the UK’s MSA Trust as a funding partner in this critical work.
Charlotte, NC, Nov. 28, 2017 – The Multiple System Atrophy (MSA) Coalition today (Giving Tuesday) announced that it has, in partnership with other MSA charities, awarded nine new research grants totaling $400,000 to scientists in pursuit of a cure for this devastating neurodegenerative disease. The money for this year’s grants was primarily donated by grass-root fundraisers, individuals affected by MSA, and by partnership with the MSA Trust. The leading nonprofit funder of global MSA research, the MSA Coalition, along with financial support from several other MSA charities, has now granted a total of $1.7 million since 2013 to fund 36 research projects aimed at improving the diagnosis and symptomatic treatment — and finding a potential cure —, for this rare and terminal disease.
This latest round of research grants was approved by the MSA Coalition’s Board of Directors following a thorough review by the Scientific Advisory Committee, through which leading scientists and clinicians from around the world participate in volunteer roles to oversee the peer-review process.
Interest in the MSA Coalition’s research grant program by the scientific community continues to grow year over year. This year, the MSA Coalition received 51 grant applications —up from 36 in 2016 —and awarded grants to nine leading scientists conducting innovative research targeting MSA. Recipients include investigators working in research laboratories in the United States as well as in Australia, Austria, Denmark, France, Germany, Italy and Sweden.
The MSA Coalition continues to fund the world’s best researchers working on the most promising scientific issues. With these new awards, the goal is to make progress in three research pathways: identifying definitive new biomarkers that might allow earlier and more accurate diagnosis of MSA; validating in a pre-clinical setting potential therapies using transgenic MSA animal models as test subjects; and pursuing increased understanding of the underlying cause of MSA through in-depth scientific studies.
“My wife was diagnosed in 2013, the same year the MSA Coalition began funding research, and the difference between then and now in the number of potential hypotheses, researchers to pursue them and collaborative efforts to bring positive news to patients like Colleen is quite significant,” notes Dr. Larry Kellerman, MSA Coalition Board Member and Co-chair of the Research Committee. “The research grants awarded by the MSA Coalition have significantly increased the level of global scientific collaboration and interest in beating this rare, terminal and sporadic disease; and thus, hope for all impacted by multiple system atrophy. It is also really exciting to see international collaboration with the MSA Trust. Together, the MSA community is stronger and this partnership further validates the rigorous vetting process of the MSA Coalition Research Grant Program.”
Highlights of the new research grants awarded by the MSA Coalition include:
- Facilitating data collection and sharing among a global network of MSA researchers. Because MSA is a rare disease, it is crucial to establish a global infrastructure to recruit adequate numbers of patients for clinical trials, to collect and share patient data and to conduct natural history studies looking for commonalities in disease characteristics and progression rates. The MSA Coalition has renewed funding for the Global MSA Registry (GLOMSAR) and Natural History Study, spearheaded by researchers at New York University’s Dysautonomia Center.
- Establishing a new and improved animal model of multiple system atrophy. There is an urgent need to develop new cell and animal models that more closely mimic human MSA to assist in finding potential new drug targets and to use as test subjects to better predict efficacy in early stage drug development. Researchers at Rush University in Chicago and Lund University (Sweden) aim at replicating both MSA-Parkinson type and MSA-Cerebellar type, respectively, in new rat models using an innovative approach of injecting an oligodendrocyte-selective, adeno-associated viral vector into rat brains.
- Seeking new genetic clues to aid in understanding multiple system atrophy. Increased understanding of the underlying cause (pathogenesis) of MSA can lead to the development of new interventions to stop or slow disease progression. Researchers at University of Tubingen (Germany) and the Medical University of Innsbruck (Austria) are collaborating to screen more than 800 genetic samples from European MSA patients using the latest Neurochip genotyping tool. This tool will enable screening for more than 450,000 genetic variants implicated in a wide range of neurodegenerative diseases. Moreover, it will help to screen for MSA patients and people at risk for MSA in the clinical setting, and thus allow a deeper insight into pathogenesis even at early stages of the disease.
- Developing a simple blood test (biomarker) for multiple system atrophy. The discovery of a biomarker to detect specific blood changes would allow patients to be diagnosed much sooner, before the disease has progressed significantly. Researchers at the University Hospital Bordeaux (France), University of Salerno (Italy) and the National Institutes of Health (USA) are collaborating to develop a blood biomarker that could diagnose MSA as well as measure disease severity and progression. This biomarker study closely relates to a previous MSA Coalition grant aimed to develop a comprehensive rationale for administering Exendin-4 as a treatment for MSA patients. (The diabetes drug Exendin-4 previously was tested in an MSA animal model with positive results. See: https://www.multiplesystematrophy.org/blog/report-msa-coalition-funded-research-exendin-4)
- Testing potential therapies for multiple system atrophy. Researchers at the Florey Institute in Melbourne (Australia) will assess the effects of two therapeutic options in MSA animal models: ceruloplasmin supplementation or iron chelation with deferiprone. Because both drugs have already been approved by the U.S. Food and Drug Administration, a successful outcome of this study could offer the possibility of proceeding quickly with clinical trials in MSA patients.
Critical funds to support these research awards came from key MSA Coalition partners and supporters including the Multiple System Atrophy Trust, the Patrick Costigan MSA Research Fund, Blandford Rees Foundation, Deborah Knutson Memorial Research Fund, Move Over MSA, Dan Cavanaugh Memorial Fund, Defeat MSA, MSA NJ, and from many dedicated families and individual donors personally affected by the disease. Prof Clare Fowler, Chairman of the MSA Trust stated “a reliable and regular source of funding is essential if we are to build up and sustain expertise in MSA research. This is something we have been trying to do in the UK over the last 10 years and for the same reason we are delighted to have been able to contribute to the MSA Coalition’s global funding round this year.”
The MSA Coalition is tremendously grateful for our strong commitment of fundraisers, donors, 15,000+ social media followers, and partner organizations, whose generous support enables us to continue funding promising MSA research around the world. This year we are thrilled to welcome the MSA Trust (UK), the leading MSA charity overseas, as a global research funding partner,” says Cynthia Roemer, MSA Coalition Chairperson.
“Since MSA is a very rare and little-known disorder, participation by our community is critical. There is another big opportunity this year for our supporters to make a difference for multiple system atrophy,” Roemer continued. “On November 28, MSA families around the world will have the opportunity to join our efforts to support more MSA research. A full 100 percent of proceeds donated online on Giving Tuesday to the MSA Holiday Hope for a Cure campaign will be earmarked for research. In addition, $150,000 in matching gift funds are available. Give $100 and it automatically turns into $200 for our organization!”
The complete list of 2017 MSA Coalition Research Grant Award projects/recipients is as follows:
- “Global MSA Registry & Natural History Study – Year 4”: Lucy Norcliffe-Kaufmann, Ph.D. (New York, NY)
- “Towards translational animal models of MSA”: Jeffrey Kordower, Ph.D. (Chicago, IL), Deniz Kirik, M.D., Ph.D. (Lund, Sweden), and Mari Savolainen, Ph.D. (Chicago, IL)
- “Pathological alpha-Synuclein in glial cytoplasmic inclusions represent a unique alpha-Synuclein strain with post translational modifications”: Chao Peng, Ph.D. (Philadelphia, PA), and Virginia Lee, Ph.D. (Philadelphia, PA)
- “Defining novel pathways for multiple system atrophy”: Manu Sharma, Ph.D. (Tubingen, Germany), Gregor K. Wenning, M.D., Ph.D. (Innsbruck, Austria), and Florian Krismer, M.D., Ph.D. (Innsbruck, Austria)
- “Plasma exosomal IRS-1pS312 as biomarker for MSA”: Wassilios Meissner, M.D., Ph.D. (Bordeaux, France), Dimitrios Kapogiannis, M.D. (Baltimore, MD), and Maria Teresa Pellecchia, M.D., Ph.D. (Salerno, Italy)
- “Proteomic analysis of glial cytoplasmic inclusions in MSA”: Thomas Wisniewski, M.D. (New York, NY), and Horacio Kaufmann, M.D. (New York, NY)
- “A novel diagnostic test for distinguishing synucleinopathies”: Gal Bitan, Ph.D. (Los Angeles, CA)
- “Towards new strategies for effective immunotherapy in Multiple System Atrophy”: Bente Pakkenberg, M.D. (Copenhagen, Denmark), and Tomasz Brudek, M.D., Ph.D. (Copenhagen, Denmark)
- “Can ceruloplasmin or metal chelation be used as a therapeutic agent in an animal model of MSA?”: David Finkelstein, Ph.D. (Melbourne, Australia), and Erin McAllum, Ph.D. (Melbourne, Australia)