MSA Coalition Research Grants – Funded Projects
Listed below are the research projects funded by The Multiple System Atrophy Coalition as part of the MSA Research Grant Program. The below projects were selected from many applications after bing reviewed and ranked by The MSA Coalition’s Scientific Advisory Board (SAB). These projects are deemed to have strong scientific merit toward accomplishing The MSA Coalition’s primary mission of identifying a cause and a cure for MSA.
2014 MSA Research Grant Award Recipients:
"Global MSA Registry & Study Group": Florian Krismer, M.D. (Innsbruck Medical University) and Lucy Norcliffe-Kaufmann, Ph.D. (New York University)
This project aims to establish the first-ever global registry dedicated to Multiple System Atrophy patients. Facilitating future worldwide clinical trials, the registry will be used to notify all patients that meet study entry criteria for clinical trials in MSA on an international scale. The registry will also provide a means for sharing anonymous patient information to define the disease specific characteristics and establish the definitive natural history of MSA. Registered patients will be followed thoroughly and periodically to identify potential biological markers of disease risk and severity in a global, worldwide longitudinal prospective study.
"Stem cell-based Therapeutics Platform for MSA": Vikram Khurana, M.D, Ph.D. (Massachusetts General Hospital)
This project aims to develop new models of Multiple System Atrophy by utilizing stem cell technology to generate human patient-derived stem cell models of the disease. This will enable the study of the biology of an individual patient’s disease in the dish by creating their neurons and oligodendrocytes and then looking for signatures of alpha-synuclein toxicity. If alpha-synuclein toxicity signatures are identified it will further assess whether it’s possible to reverse this toxicity with genes and small molecules. This work may lead to a new way of testing and discovering potential therapies for MSA beyond the current mouse models of the disease.
"Mechanisms of Excessive Daytime Sleepiness and Sleep Related Respiratory Dysfunction in MSA": Eduardo Benarroch, M.D. (Mayo Clinic Rochester)
Excessive daytime sleepiness and sleep related respiratory disorders such as sleep apnea and laryngeal stridor are prominent symptoms in patients with Multiple System Atrophy. This study aims to uncover the underlying mechanisms of these sleep disorders through pathological studies. Understanding the underlying causes of sleep disorders associated with MSA can provide rationale for development of pharmacological approaches for treatment of these conditions. This can potentially lead both to improvement of quality of life and prevention of premature death in MSA patients.
"Peripheral delivery of brain-targeted neurosin as a novel treatment for MSA": Eliezer Masliah, M.D. (University of California San Diego)
Recent studies suggest that abnormal accumulation of the protein alpha-synuclein in brain cells leads to cellular dysfunctional and neurodegeneration in Multiple System Atrophy and it is increasingly evident that the toxicity of extracellular alpha-synuclein might be related to its ability to be taken up by neighboring cells and act in a prion-like manner. This behavior could be a key event in the origin and progression of the disease.
Compounds that reduce intracellular alpha-synuclein aggregation have received significant attention in recent years but the possibility of reducing the spread of alpha-synuclein from cell to cell by degrading extracellular alpha-synuclein has not been explored in such depth. This project aims to increase the brain levels of an extracellular alpha-synuclein degrading enzyme in mouse models of MSA by means of peripheral gene therapy and targeted delivery to the central nervous system (CNS). Delivery of brain targeted neurosin into the CNS might represent a potential therapeutic option for neurodegenerative disorders including Multiple System Atrophy.
"Biomarker Development in MSA": Roy Freeman, M.D. (Beth Israel Deaconess Medical Center)
There is currently an unmet need for a biomarker in Multiple System Atrophy. A reliable biomarker could contribute to the diagnosis, treatment and disease modification of MSA by improving diagnostic accuracy, defining disease progression and providing an objective measure of the response to disease modifying interventions.
Successful development of a biomarker for MSA would therefore assist in the evaluation and enhance the efficacy of drugs or other interventions that have neuroprotective qualities and offer the possibility of slowing, halting or reversing the rapid progression of MSA.
This study in human subjects, will determine whether alpha-synuclein deposits in cutaneous autonomic nerves is a valid biomarker for multiple system atrophy.