A cancer drug for Parkinson’s Disease?

Published: November 4th, 2015

Category: UAD Student Blog

Pills Question MarkParkinson’s Disease (PD) is a chronic and progressive neurodegenerative disorder that results in the loss of dopaminergic neurons within the brain. This leads to symptoms such as tremors, bradykinesia, rigidity, and postural instability. Currently there is no cure for Parkinson’s Disease, but there are treatments that can help manage the symptoms and slow the progression (National Institutes of Health, 2015). Researchers at Georgetown University Medical Center (GUMC) concluded a study in May of this year that tested the effect of Nilotinib on twelve individuals diagnosed with Parkinson’s Disease (PD), Parkinson’s Disease Dementia (PDD) and Lewy BodyDementia (LBD). The aim of this clinical trial was to test the safety and efficacy of the drug (Pagan, 2015).

Nilotinib is an FDA-approved drug for leukemia that forces cancer cells into autophagy, a process that leads to cell death. According to Charbel Moussa, MD, PhD, “It appears that in smaller doses once a day, Nilotinib turns on autophagy for about four to eight hours – long enough to clean out the cells without causing cell death.” It is on this premise that the clinical trial was based. Nilotinib, in smaller doses, was used to act as a “garbage disposal,” clearing out the toxic proteins within the brain. During the six-month trial, patients were given Nilotinib in escalating doses (150-300mg) daily. Ten of the twelve participants reported meaningful changes during the trial period, including improved cognition, motor skills, and non-motor function. One participant of the study regained the ability to walk. Three others regained their ability to engage inconversation (Georgetown University Medical Center, 2015). Nilotinib led to positive changes in CSF biomarkers of Parkinson’s, including alpha-synuclein,amyloid beta-40/42, and dopamine, as well as statistically significant changes in total Tau and p-Tau. These improvements receded within weeks of the trial’sconclusion (Pagan, 2015).

While these preliminary results create some hope for patients, their families, and professionals, further research is indicated. The primary goal of the study was to evaluate the safety of the drug over a six-month period. Thus the drug’s impact on specific symptoms (speech, swallow, cognition, movement, gait and posture) and on patients’ disease severity needs to be thoroughly examined. As well the sample size was small with only 12 participants. So even though GUMC’s results are promising, larger sample size, over a longer period of time, utilizing a randomized control trial is needed to truly determine the impact Nilotonib can have on PD, PDD, and LBD.


Duffy, J.R. (2013). Motor Speech Disorders: Substrates, Differential Diagnosis, andManagement (3rd ed.). St. Louis, MO: Elsevier Mosby.

Georgetown University Medical Center (2015). Cancer Drug Improved Cognition and Motor Skills in Small Parkinson’s Clinical Trial. Retrieved from https://gumc.georgetown.edu/news/Cancer-Drug-Improved-Cognition-and-Motor-Skills-in-Small-Parkinsons-Clinical-Trial.

National Institute of Health (2014) Nilotinib in Cognitively Impaired Parkinson Disease Patients. Retrieved from https://clinicaltrials.gov/ct2/show/study/NCT02281474.National Institutes of Health(2015).

NINDS Parkinson’s Disease Information Page.Retrieved from http://www.ninds.nih.gov/disorders/parkinsons_disease/parkinsons_disease.htm

Pagan, F.L. Nilotinib significantly alters blood and CSF α-Synuclein and p-Tau levels,inhibits dopamine breakdown and increases neuro-restorative markers in an open-labelled Parkinson’s disease with dementia and Lewy body dementia trial. ProgramNo. 12.01. 2015 Neuroscience Meeting Planner. Washington, DC: Society forNeuroscience, 2015. Online.