Support for Multiple Systems Atrophy (MSA) Research

GUMC Translational Neurotherapeutics Program (TNP) Hero Image

Georgetown University Medical Center's Translational Neurotherapeutics Program (TNP) conducts cutting-edge research that improves treatment options for neurodegenerative diseases and provides unique, state-of-the-art clinical services for those affected by Multiple Systems Atrophy (MSA) and other related conditions. Charbel E-H Moussa, MBBS, Ph.D. is the director of Georgetown's Laboratory of Dementia and Parkinsonism, clinical research director of the TNP (a National Parkinson Foundation Center of Excellence), and an assistant professor in the Department of Neurology at Georgetown University Medical Center.

Charbel E-H Moussa, MBBS, Ph.D.

What are we doing?

Nilotinib is an anti-cancer drug. Moussa has performed several studies to repurpose this drug for neurodegenerative diseases, providing compelling evidence that Nilotinib is highly useful in eliminating toxic protein accumulation and reducing protein secretion via a process called autophagy (self-eating). Moussa's findings with Parkinson's disease (PD) and Lewy Body Dementia (LBD) have yielded unprecedented and remarkable improvement in cognitive and motor functions in both pre-clinical mouse models and a proof-of-concept study in humans.

With the discovery that Nilotinib can clear neurotoxic proteins at a safe and much lower dose than that which is 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 (AD), LBD, Amyotrophic Lateral Sclerosis (ALS), Huntington's disease (HD), and dementia.

 

What's next?

Philanthropic support in the amount of $300,000 will allow Moussa and the Translational Neurotherapeutics Program to execute the Phase I study to evaluate the effects of Nilotinib on motor, cognitive, and autonomic symptoms of MSA. The study will establish the safety and tolerability of Nilotinib in MSA, determine drug effects on disease-related biomarkers, and demonstrate potential disease-modifying effects in MSA. The study will include 12 patients and will lead to a larger Phase II trial. To date, a leadership gift of $50,000 has reduced our need to $250,000 to start the trial.

How can I help?

Commitments at any level have both immediate and measurable impact. On behalf of Moussa and the program, 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. For more information about supporting this research, please contact Kristina Madarang, associate director of Development, at (202)687-2464 or Kristina.Madarang@georgetown.edu. You may also contribute online. Simply use the "Other Designation" option and type in "Nilotinib for MSA."