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Nilotinib for Parkinson’s disease

Researchers report on the results of a randomized clinical trial of Nilotinib for Parkinson’s disease.

Parkinson’s disease is one of the most common neurodegenerative diseases. The disease currently has no cure and symptoms are managed through medication and various forms of therapy. Based on the nature of Parkinson’s disease, researchers have begun to study the drug nilotinib to determine whether it can be used as a treatment option for Parkinson’s. Nilotinib is a drug that has already been determined to be safe for use in the treatment of leukemia. Previous studies appear to suggest that nilotinib could increase dopamine metabolism and could also possibly treat the motor and nonmotor symptoms of Parkinson’s disease.

In a study published in JAMA Neurology, a group of researchers aimed to investigate how safe nilotinib was and what its effects were on biomarkers in patients with Parkinson’s disease. The researchers performed a randomized clinical trial to determine the safety of nilotinib, how tolerable it is, and how the drug behaves in patients with Parkinson’s. They also tested the effect of this treatment on two biomarkers – HVA and DOPAC. In addition, the researchers aimed to investigate the drug’s effects on the α-synuclein protein and what effects the drug would have on motor and nonmotor symptoms of Parkinson’s. The patients who participated in the study were either treated with the placebo, nilotinib 150-mg, or nilotinib 300-mg.

The investigation into the drug’s actions in the body revealed that small amounts of nilotinib were able to be detected in the cerebrospinal fluid at the 12-month mark. The researchers also found a significant increase in dopamine metabolism – demonstrated by an increase in metabolites of dopamine present in the cerebrospinal fluid – that was seen at lower doses of nilotinib. A reduction in complexes of α-synuclein was seen, which the researchers believe may lead to improved activity of neurons that use dopamine as a neurotransmitter. In terms of the potential effect on motor and nonmotor symptoms of Parkinson’s, there were no significant differences seen between the three treatment groups.

The researchers note that their study was underpowered and was performed in only one centre that treated Parkinson’s patients, which is potentially a problem as various treatment centres could have different pain management and treatment techniques that could interfere with the results. Nonetheless, the primary objective of the study was met in that nilotinib was deemed to be “reasonably safe, tolerated, and detected in the cerebrospinal fluid of patients with Parkinson’s disease”. More research into the effects of nilotinib is needed, but the results of this study will help to guide the development of future studies that specifically target the effects of nilotinib as a therapy option for patients with Parkinson’s.


Written by Haritha Thevar, BSc



Pagan, F., Hebron, M., Wilmarth, B., Torres-Yaghi, Y., Lawler, A., & Mundel, E. et al. (2019). Nilotinib Effects on Safety, Tolerability, and Potential Biomarkers in Parkinson Disease. JAMA Neurology. doi: 10.1001/jamaneurol.2019.4200

Understanding Parkinson’s. (2019). Retrieved 22 December 2019, from

Image by Steve Buissinne from Pixabay



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