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

Alan R. Jacobs, MD


August 25, 2016

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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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