Nilotinib Shows Early Safety, Efficacy in Parkinson' Dementia and Dementia With Lewy Bodies

Alan R. Jacobs, MD, August 25, 2016

This is the Medscape Neurology Minute. I'm Dr Alan Jacobs.

Researchers from Georgetown University Medical Center in Washington, DC, have published an open-label, proof-of-concept study to evaluate the safety and tolerability of nilotinib (Tasigna®, Novartis), an FDA-approved medication for adults with chronic myeloid leukemia, in subjects with Parkinson's dementia or dementia with Lewy bodies.^[1]

Twelve subjects were randomized into 150-mg or 300-mg groups and received nilotinib orally every day for 24 weeks.

Results showed nilotinib to be safe and well tolerated. Serum and spinal fluid biomarker analysis revealed a steady doubling of homovanillic acid, such that most participants were able to stop or reduce the use of their dopamine replacement therapies. Moreover, the level of the Parkinson's disease-related oxidative stress marker DJ-1 was reduced more than 50% after nilotinib treatment, consistent with less dopaminergic neuron death. Levels of three markers of cell death—neuron-specific enolase, S100B, and tau—were significantly reduced in cerebrospinal fluid, suggesting reduced neuronal cell death. Even cerebrospinal fluid levels of alpha-synuclein were attenuated significantly.

The authors concluded that the evaluation of the safety and efficacy of nilotinib in larger randomized, double-blind, placebo-controlled trials is clearly warranted.

This has been the Medscape Neurology Minute. I'm Dr Alan Jacobs

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