Study Rationale:
Nilotinib, a drug approved for chronic myelogenous leukemia, inhibits the protein c-Abl. Higher brain levels of c-Abl are associated with Parkinson's disease (PD), and laboratory studies have linked c-Abl to cellular pathways believed to underlie PD. Nilotinib has also shown protective effects in PD preclinical models. Additionally, a small Phase I trial reported nilotinib was well-tolerated and led to improvements in motor skills and cognitive function in participants with advanced Parkinson's disease and Lewy body dementia. However, as the trial was open label (no placebo control group), additional research is needed to understand the potential clinical benefit of nilotinib for PD.
Hypothesis:
The planned trial will test the hypothesis of whether nilotinib is safe and tolerable and will explore potential clinical benefits for people with PD.
Study Design:
The Michael J. Fox Foundation is partnering with The Cure Parkinson's Trust and the Van Andel Research Institute to design a randomized, double-blind, placebo-controlled Phase II trial to learn more about nilotinib's safety and impact on PD. The current grant supports a planning committee of leading PD clinicians and researchers to design the study. Members of the planning committee include: Patrik Brundin, MD, PhD of Van Andel Research Institute; Helen Matthews and Richard Wyse of Cure Parkinson's Trust; patient representative Gary Rafaloff; Christopher Coffey, PhD; Ted Dawson, MD, PhD; Kalpana Merchant, PhD; David Weiner, MD; Michael Schwarzchild, MD, PhD; David Simon, MD, PhD; Tanya Simuni, MD; and Charles Venuto, PharmD.
Impact on Diagnosis/Treatment of Parkinson's Disease:
If nilotinib is found to be safe in people with Parkinson's and shows continued promise in easing PD symptoms or slowing its progression, it could become a promising treatment for patients.
Next Steps for Development:
MJFF and its partners seek to launch the trial by mid-2017.
Additional Support:
The Michael J. Fox Foundation would like to acknowledge the generous contribution of the Demoucelle Parkinson Charity as a lead supporter providing funding for this phase of the project.