Global efforts to achieve a greater understanding of multiple system atrophy, a rare and devastating neurodegenerative disease, have been enhanced by ten research projects receiving awards in 2016. The MSA Coalition Research Grant Program began awarding funds in 2014 and has now funded a total of 27 research projects around the world.
Charlotte, NC – October 3, 2016 (World MSA Day): The Multiple System Atrophy Coalition, the leading nonprofit funder of multiple system atrophy (MSA) research, is excited to announce its Board of Directors has awarded $532,000 to 10 research projects this year. With the addition of this year’s award total, the MSA Coalition has directed over $1.25 million in its three-year research granting history to scientists around the world in pursuit of a cure for this devastating neurodegenerative disease. The 2016 research projects and recipients are as follows:
- “Global MSA Registry & Natural History Study”: Lucy Norcliffe-Kaufmann, Ph.D. (New York University) and Gregor K. Wenning, M.D., Ph.D. (Innsbruck Medical University, Austria)
- “The role of hemoglobin overexpression in molecular pathology of MSA”: Michael Janitz, M.D., Ph.D. (University of New South Wales, Australia) and Ronald Melki, Ph.D. (Paris-Saclay Institute of Neuroscience, France)
- “COQ2 mutation and methylation dysfunction leading to alpha-synuclein pathology”: W. Scott Kim, Ph.D. (University of Sydney, Australia), Glenda Halliday, Ph.D. (University of Sydney, Australia) and Poul Jensen, M.D., Ph.D. (University of Aarhus, Denmark)
- “Unravelling the mechanism of alpha-synuclein seeding in oligodendrocytes”: Maria Xilouri, Ph.D. (Biomedical Research Foundation of the Academy of Athens, Greece)
- “Targeting alpha-synuclein pathology with the molecular tweezer CLR01 in MSA: optimization of drug delivery and biochemical analysis”: Nadia Stefanova, M.D, Ph.D. (Innsbruck Medical University, Austria) and Gal Bitan, Ph.D. (UC Los Angeles)
- “GRK2 targeted knock-down as therapy for multiple system atrophy”: Erwan Bezard, Ph.D. (University of Bordeaux, France)
- “Nilotinib for treating MSA: a preclinical proof of concept study”: Pierre-Olivier Fernagut, Ph.D. (University of Bordeaux, France) and Wassilios Meissner, M.D., Ph.D. (University of Bordeaux, France)
- “A serum miRNAs signature as potential biomarker for MSA”: Anna Maria Vallelunga, MSc (University of Salerno, Italy) and Maria Teresa Pellecchia, M.D., Ph.D. (University of Salerno, Italy)
- “Inside the gait – a new era on the horizon for atypical parkinsonian disorders”: Gregor K. Wenning, M.D., Ph.D. (Innsbruck Medical University, Austria)
- “Toward the in situ proteome of normal and pathologic alpha-synuclein in human neurons and glial cells”: Vikram Khurana, M.D., Ph.D. (Brigham and Women’s Hospital, Boston)
A layman’s summary with more information about all funded MSA research projects can be found at www.multiple-system-atrophy.org.
“Never did I imagine we would one day be able to say we have awarded over $1 million in research grants”, said Judy Biedenharn, Board Chair of The Multiple System Atrophy Coalition. “When we started our research grant program three years ago we had a little over $200,000 available. Just 9 researchers sent in proposals that year from which we funded 5 projects. It’s really exciting to see the continued growth of this research program which has gone hand in hand with the increased fundraising efforts of so many families. Donations large and small have made all of this work possible.”
This year 36 letters of intent or project pre-proposals were received and reviewed by the MSA Coalition’s Scientific Advisory Board. The board, made up of leading scientists and clinicians in MSA research, ensures the MSA Coalition identifies those projects that are most likely to move the understanding of multiple system atrophy and its underlying causes forward. Out of those 36 pre-proposals 22 were then chosen and invited to submit full proposals to the final competition.
“As our research program has expanded we have had to change our methodology to keep up the pace. In early 2015 we consulted with the Michael J. Fox Foundation and they very generously allowed us to adopt their grant peer review process”, stated Pam Bower, Board of Directors Secretary and Research committee co-chair. “Having this framework to follow has helped us ensure every proposal is vetted appropriately by three totally independent reviewers with no connection to the project. Maintaining this high standard of fairness and transparency is very important to us and the end result of being able to provide research funding to 10 high merit projects is a very satisfying conclusion to a many months long process.”
Funded projects include investigation of new biomarkers that might allow earlier and more accurate diagnosis, pre-clinical validation of potential therapies using transgenic MSA mouse models as test subjects as well as in-depth scientific studies pursuing the underlying cause of MSA. Awards were granted to investigators working in neurodegeneration disease research labs around the world including the United States, Australia, Austria, Denmark, France, Italy and Greece.
“The sense of urgency felt by MSA patients and their loved ones is a big motivating factor for all who participate in the research grant process”, noted Larry Kellerman, Board Member and Caregiver Representative. “Every board member has a personal connection to the disease and represents thousands more anxiously waiting to receive some good news. The funded projects are seen as the best approach to arriving at positive results and thus are a reason to hope.”
Besides performing the research, the recipients of the grant dollars are also required to submit an interim and final report on their project. Those reports provide a window into the progress being observed by the researchers and can lay the groundwork for other researchers and for future funding opportunities. Since these awards are considered seed grants they may lead their recipients to submit proposals to other, larger funding agencies to continue their work.
The Multiple System Atrophy Coalition first established a dedicated MSA research fund in 2011. In 2013, the organization launched its MSA Research Grant Program and recruited a world class Scientific Advisory Board (SAB) made up of leading scientists and clinicians in the MSA field. The goal of the MSA Research Grant Program is to identify and fund MSA research projects that have potential to uncover the cause, discover disease modifying treatments, and hopefully lead to a cure. Through a rigorous peer review process, the SAB ensures that The MSA Coalition is funding the most promising next steps that can lead to breakthroughs in MSA research. At present, a minimum of 75% of every donation received by The MSA Coalition goes directly into the research fund. The remaining 25% goes toward much needed support and education programs including a toll-free support hotline (1-866-737-5999), educational materials, an annual patient/caregiver conference (also live-streamed on the internet), continuing medical education about MSA for healthcare professionals as well as important advocacy and awareness initiatives.