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Michael J. Fox Foundation Announces Award to Drive Phase IIa Clinical Trial Investigating Potential Safety and Benefit of Nilotinib in Parkinson's Disease

  • Trial aims to establish safety of long-term nilotinib use in people with Parkinson's disease; recruitment to begin in September 2017
  • Endpoints to include symptomatic response and biological measures of drug activity
  • Patients and clinicians urged to use caution before adding a repurposed therapy such as nilotinib to treatment regimens

NEW YORK (July 28, 2017) -- The Michael J. Fox Foundation for Parkinson's Research (MJFF) today announced U.S. Food and Drug Administration (FDA) approval of a multicenter, double-blind, randomized, placebo-controlled Phase IIa clinical trial to test safety and tolerability of nilotinib (Tasigna) in Parkinson's disease (PD). The trial is designed to investigate the potential of repurposing nilotinib, an FDA-approved treatment for chronic myelogenous leukemia, for use in Parkinson's disease.

The trial will be led by prinicipal investigator Tanya Simuni, MD, professor of neurology and head of the division of movement disorders at Northwestern University Feinberg School of Medicine, and carried out at 25 clinical sites across the United States through the Parkinson Study Group, the largest not-for-profit scientific network of Parkinson's disease centers in North America.

Nilotinib inhibits the activity of c-Abl, a protein that has been linked to cellular pathways associated with Parkinson's disease. Preliminary data from a small open-label Phase I clinical trial evaluating the safety and tolerability of nilotinib in people with advanced Parkinson's showed potential benefit in PD. The Phase IIa trial aims to expand on these preliminary safety findings, and assess potential dosing range, to better understand the implications of nilotinib's long-term use in Parkinson's. The study also will further explore nilotinib's potential to treat symptoms or to slow or stop disease progression (something no current PD treatment has been proven to do).

"The Michael J. Fox Foundation is dedicated to pursuing every research avenue that could accelerate progress in Parkinson's drug development," said Todd Sherer, PhD, chief executive officer of MJFF. "While much work remains to be done to establish if nilotinib is a promising therapy in Parkinson's, we hope to contribute to understanding of fundamental questions around its tolerability, dosing, and whether and at which stage of Parkinson's it may work."

Tanya Simuni added: "As we further explore the potential safety and tolerability of nilotinib, we look forward to reporting our data and findings to researchers and patients. In the meantime, we continue to urge patients and neurologists not to add nilotinib to their Parkinson's treatment regimens until more is understood about the safety and possible efficacy of the drug in PD."

Trial Design Aims to Yield Definitive Results
In the trial, 75 people with moderate to advanced Parkinson's disease will be randomly assigned to receive daily doses of 150 mg of nilotinib, 300 mg of nilotinib or placebo for six months. Participants will be closely monitored throughout the dosing period and for 8 weeks after all treatments are stopped to gain insights into nilotinib's safety and tolerability, and to determine the maximum dose tolerated. If outcomes provide conclusive safety data, a second funded trial will test nilotinib in a group of 60 people with early PD for 12 months at the highest tolerated daily dose of niltotinib versus placebo.

Trial participants will complete motor, cognitive and biological (i.e., blood and spinal fluid) tests, providing data for investigators to evaluate the potential impact of nilotinib on the target pathway, as well as on c-Abl biology and Parkinson's symptoms and/or disease progression.

MJFF has collaborated with the Van Andel Research Institute (VARI) of Grand Rapids, Michigan, and The Cure Parkinson's Trust (CPT) of London, United Kingdom, on the clinical development of nilotinib toward the launch of this clinical trial. Partial funding for the trial has been generously provided through an anonymous leadership gift from a family living with Parkinson's disease, as well as contributions from the Demoucelle Parkinson Charity (Belgium) and The Parkinson Alliance (Kingston, New Jersey). Study leadership is working to secure in-kind donation of drug and placebo for use in the trial.

Recruitment is anticipated to begin in September 2017. Patients interested in participating should create a profile on Fox Trial Finder ( to be notified when enrollment opens if they are a likely match for this study or others in need of research volunteers.

The Promise of Repurposed Therapies to Accelerate Parkinson's Drug Development
Nilotinib is one of many examples of a repurposed (or repositioned) drug for Parkinson's. Repurposing is a promising and relatively efficient way to seed the pipeline of drugs by taking an exisiting medicine approved by the FDA to treat one condition and using it to treat another. Repurposed drugs can usually skip Phase I clinical testing because they are already proven safe in human volunteers. However, Phase II and Phase III trials are required to rigorously evaluate the safety, tolerability and efficacy of these treatments in people with Parkinson's.

Repurposing c-Abl compounds, such as nilotinib, within the context of Parkinson's disease is an area of increasing research focus. The MJFF-funded nilotinib trial joins others currently in the recruiting and planning stages. Multiple concurrent trials such as these can provide more data to speed conclusions about a drug's therapeutic potential.

It is important for researchers to conclusively demonstrate repurposed therapies are effective in Parkinson's, and more critically, that they are safe for people with PD to take long term. Patients and clinicians are urged to wait for additional data before adding nilotinib or any other repurposed treatments to their regimen.

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Media Contact:
Amy Schultz

About The Michael J. Fox Foundation for Parkinson's Research
As the world's largest nonprofit funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, industry leaders, government research funders and regulators, clinical trial participants, donors and volunteers. In addition to funding more than $700 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. The Foundation increases the flow of participants into Parkinson's disease clinical trials with its online tools Fox Trial Finder and Fox Insight; sponsors a landmark, international study to find reliable and consistent biomarkers of Parkinson's progression; promotes Parkinson's awareness through high-profile events and outreach; advocates for state and federal policies that support medical research and increased access to health care; and coordinates the involvement of thousands of Team Fox members and grassroots volunteers around the world.

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