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Proposal for Funding in Support of Multiple Systems Atrophy Research at Georgetown University Medical Center Georgetown University Medical Center’s Translational Neurotherapeutics Program (TNP) conducts cutting-edge translational research that improves treatment options for neurodegenerative diseases and provides unique, state-of-the-art clinical services for individuals affected by Multiple Systems Atrophy (MSA) and other related conditions. Nilotinib is an anti-cancer drug that degrades misfolded and toxic protein debris and prevents the accumulation of alpha-Synuclein in the central nervous system and peripherally in other body organs. Charbel Moussa, MD, PhD performed several studies to repurpose this drug for neurodegenerative diseases, providing compelling evidence that Nilotinib is highly useful in eliminating toxic protein accumulation within the cell, thus reducing protein secretion into the extracellular space, via a process called autophagy (self-eating). Support in the amount of $200,000 will allow Dr. Moussa and the Translational Neurotherapeutics Program to execute a phase I study to evaluate the effects of Nilotinib on motor, cognitive and autonomic symptoms in MSA. This study aims to establish the safety and tolerability of Nilotinib in MSA, determine drug effects on disease-related biomarkers like alpha-Synuclein and dopamine, and demonstrate potential disease-modifying effects in MSA. This study will include a total of 12 patients, and would ideally lead to a larger phase II trial. Dr. Moussa’s findings with Parkinson’s disease (PD) and Lewy Body Dementia (LBD) to date have yielded unprecedented and remarkable improvement in cognitive and motor functions in both pre-clinical mouse models and clinical trials in humans. With the discovery that Nilotinib can clear neurotoxic proteins at a safe and much lower dose than the dose prescribed in cancer, Nilotinib could be the key to combatting and modifying the whole spectrum of these conditions, from PD to MSA, Alzheimer’s disease, LBD, Amyotrophic Lateral Sclerosis (ALS), and dementia. Commitments at any level have measurable and immediate impact. On behalf of Dr. Moussa and the TNP, whose work will be fulfilled by your generosity, we are grateful to have you as a partner in our commitment to human health, excellence in research, and victory over MSA. TNP Clinical Research Director Charbel Moussa, MD, PhD, is the Director of Georgetown’s Laboratory of Dementia and Parkinsonism, Clinical Research Director of the Translational Neurotherapeutics Program (a National Parkinson Foundation Center of Excellence), and an assistant professor in the Department of Neurology at Georgetown University Medical Center. He has 58 publications and is an expert in the topics of neuroscience, neurology, neurological diseases, medical neurosciences, neurodegeneration, neuroimaging, Alzheimer’s disease, aging, neurobiology, tauopathies, amyloid, and neuroprotection. For more information about supporting cutting-edge translational Multiple Systems Atrophy research, please contact Kristina Madarang, Assistant Director of Development, at 202-687-2464 or [email protected]. gumc.georgetown.edu