In July, The Michael J. Fox Foundation for Parkinson's Research announced approval of a multicentre, double-blind, randomised, placebo-controlled Phase IIa clinical trial to test safety and tolerability of nilotinib (Tasigna) in Parkinson's (PD). The trial has been designed to investigate the potential of repurposing nilotinib, an FDA-approved treatment for chronic myelogenous leukemia, for use in PD.

MJFF has collaborated with the Van Andel Research Institute (VARI) of Grand Rapids, Michigan, and The Cure Parkinson's Trust with the clinical development of nilotinib toward the launch of this clinical trial. The trial is now recruiting and is being 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 centres in North America.

Nilotinib inhibits the activity of c-Abl, a protein that has been linked to cellular pathways associated with the progession of Parkinson's. 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. The Phase IIa trial aims to expand on these preliminary safety findings, and assess potential dosing range and 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.

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