Phase 2 Trial Testing Leukemia Treatment, Nilotinib, in Range of Parkinson’s Patients

People with Parkinson’s disease are being recruited for a new clinical trial testing nilotinib, an FDA-approved leukemia treatment, in a range of patients.

The two-part Phase 2 trial (NCT03205488), called NILO-PD, is a randomized, double-blind, and placebo-controlled study taking place at 25 sites across the U.S. Part one will evaluate the safety and tolerability of different dosing levels of oral nilotinib (150 mg or 300 mg given daily) or placebo in about 75 people with moderate to advanced Parkinson’s symptoms for 8.5 months.

Depending on results, another 60 patients with early disease symptoms will be recruited into part two. Patients will receive either the optimal dose determined from the trial’s first phase or placebo, and be regularly evaluated for 14.5 months.

Treatment tolerability and safety at six and 12 months are the trial’s main goals, but patients will also be assessed for the treatment’s potential to slow disease progression.

Nilotinib, available as a treatment for chronic myelogenous leukemia under the brand name  Tasigna, is a tyrosine kinase inhibitor that works by blocking a protein called BCR-ABL, whose activity is uncontrolled in cancer cells and leads to cell proliferation. Previous studies have shown that this protein can accumulate in the brain and is linked to several pathways — such as oxidative stress and  alpha-synuclein-induced neurodegeneration — relevant in Parkinson’s disease.

Results of a small proof-of-concept Phase 1 trial (NCT02281474), performed at Georgetown University, found two different doses of nilotinib — 150 mg and 300 mg – were well-tolerated by advanced Parkinson’s patients. Importantly, the results also suggested that the therapy may improve patients’ motor skills and cognitive abilities.

The study “Nilotinib Effects in Parkinson’s disease and Dementia with Lewy bodies” was published in the IO Press Journal in 2016.

The new trial, supported by a Michael J. Fox Foundation research grant, aims to further investigate those findings. It is led by Tanya Simuni, director of the Parkinson’s Disease and Movement Disorders Program at Northwestern University, working with the nonprofit Parkinson’s Study Group.

Specifically, researchers want to determine nilotinib’s long-term safety and effectiveness in easing symptoms. Efficacy will be assessed after six months using the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) motor score.

Enrollment information for this study, which runs through October 2020, is available by clicking here.

This study is an example of the potential of repurposing FDA-approved drugs for other diseases other than original. This can lead to faster approved therapies, since repurposed therapies have already been tested for safety and tolerability in Phase 1 trials with human volunteers.

However, the therapy’s efficacy in a new disease setting needs to be evaluated in well controlled Phase 2 and 3 clinical trials.

Researchers at the University of Rochester Medical Center’s Clinical Trials Coordination Center (CTCC) are also helping to lead the Phase 2 study.