AANS Neurosurgeon | Volume 26, Number 2, 2017

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Cancer Drug Improved Cognition and Motor Skills in Small Parkinson’s Clinical Trial

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Researchers from Georgetown University Medical Center recently reported that an FDA-approved drug for leukemia improved cognition, motor skills and non-motor function in patients with Parkinson’s disease and Lewy body dementia in a small phase-I clinical trial. The six-month, dose-escalating study of nilotinib (a treatment for typically used for chronic myelogenous leukemia or CML) showed benefit for all study patients who completed the trial (11 of 12), with 10 patients reporting meaningful clinical improvements. Patients also showed positive changes in relevant cerebrospinal fluid (CSF) biomarkers of Parkinson’s disease — alpha-synuclein (?-synuclein), amyloid beta-40/42 (Abeta-40/42) and dopamime — with statistically significant changes in total Tau and p-Tau. Prior studies show that ?-synuclein and Abeta 40/42 in CSF are decreased as Parkinson’s disease worsens, while Tau and p-Tau are increased in CSF with the onset of dementia. “The changes in Tau, p-Tau, ?-synuclein and Abeta-40 and 42 in spinal fluid suggest the clearance of toxic proteins in the brain,” said the study’s lead author. To read more about this study, click here.

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