Nilotinib by Novartis is a potential new treatment for Parkinson’s disease (PD).

Nilotinib, under the brand name Tasigna, is used to treat adults with a blood cancer called Philadelphia chromosome positive chronic myeloid leukemia (CML).  It was approved by the U.S Food and Drug Administration (FDA) in 2010 for this form of leukemia, and has been similarly authorized in the European Union since 2007.

How nilotinib works

Nilotinib is a type of biological therapy called a protein tyrosine kinase inhibitor (TKI). Tyrosine kinases are enzymes that act as chemical messengers, which can stimulate cancer cell growth.

In Parkinson’s disease, nilotinib may work to eliminate toxic proteins that build up in the brain by activating a mechanism in brain cells that serves as a sort of garbage disposal unit.

Studies with nilotinib

Researchers from Georgetown University Medical Center (GUMC) conducted a Phase 1 clinical trial (NCT02281474) that evaluated the effects of nilotinib in 11 patients with Parkinson’s and Lewy body dementia.

Results from this study suggest that the drug can safely treat both motor and cognitive problems in patients with moderately advanced Parkinson’s and mild to severe cognitive impairment.

The six-month trial reported positive outcomes in all the 11 people treated with nilotinib, with 10 patients reporting significant clinical improvement. Disease biomarkers in the cerebrospinal fluid were also evaluated, and positive changes were found for alpha-synuclein (α-synuclein), amyloid beta-40/42 (Abeta-40/42), dopamime, and tau protein, a sign of toxic protein clearance from the brain.

The results also showed that the production of dopamine increased in several patients, and that treatment discontinuation led to a deterioration in cognitive and motor skills even with levodopa therapies. Used at lower doses than for cancer patients (150 or 300 mg once daily), nilotinib seemed to improve symptoms over the 24-week treatment period, without causing serious adverse reactions.

GUMC researchers are currently conducting a larger Phase 2 clinical trial (NCT02954978), again evaluating the drug in Parkinson’s patients. They are working with colleagues at the MedStar Georgetown University Hospital, where the trial is taking place. Novartis is providing both nilotinib and a placebo.

The study will include 75 Parkinson’s disease patients who will be randomly divided into three groups. Patients in two of the groups will receive different doses of nilotinib — either 150 mg or 300 mg — while those in the third group will receive placebo. Its primary objective is to evaluate the safety and tolerability of nilotinib in patients with mid-stage Parkinson’s disease over the course of 12 months. Eligible patients will be able to take part in a one-year follow-up trial designed to evaluate the drug’s long-term effects. In the follow-up study, patients will receive either 150mg or 300mg of nilotinib. Researchers will also examine the impact that low doses have on disease biomarkers and patient outcomes.

This Phase 2 study is expected to end in July 2020.

Researchers at GUMC are also conducting a clinical trial (NCT02947893) evaluating nilotinib in people with mild to moderate Alzheimer’s disease.

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