Download Proposal for Funding in Support of Multiple Systems

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