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Cancer Drug, Nilotinib, May Slow Progression of Parkinson’s

Nilotinib, a cancer drug approved for treating some kinds of leukemia, may hold promise as a possible treatment to slow the progression of Parkinson’s disease (PD).1 Research to study the effectiveness of this drug is now ongoing.

Parkinson’s treatments including medications, Deep Brain Stimulation (DBS), and exercise, currently address PD symptoms. There is currently no cure for Parkinson’s. If this drug proves to be efficacious – successful in producing a change in the chemicals in the brain – it may be the first time we see a possible reduction in the disease process.

How does nilotinib work?

The initial 2015 study suggests that nilotinib works by “eliminating toxic proteins that build up in the brain of people with Parkinson’s and Alzheimer’s. The drug works by degrading proteins in the brain.

Early studies showed nilotinib to be effective in curing or slowing progression of Parkinson’s in mice. With further research, the drug may prove to be successful in people with multiple kinds of neurodegenerative brain disorders. The next phase of the nilotinib clinical trials were delayed by disagreements in study protocols. This has now been resolved.

Research studies currently ongoing

Current studies at Georgetown University Medical Center in Washington DC are evaluating nilotinib and placebos in people with PD and with Alzheimer’s.1 The teams are looking to see if those who receive the drug demonstrate slowed disease progression. The FDA has provided input into the study design to evaluate the whether the drug works. It will be a multi-center, double-blind, randomized, placebo-controlled clinical trial.

Nilotinib, also known by the brand name Tasigna, may prove useful in treating both the motor and cognitive components of PD. People with Parkinson’s, Dementia with Lewy bodies, and Alzheimer’s may all benefit from the changes in brain chemistry triggered by nilotinib.2

Nilotinib, a small-molecule tyrosine kinase inhibitor, works using a process called autophagy.3 It empties waste and toxic proteins from inside cells, preventing accumulation and possibly causing the cell’s death. The mechanism of action is as an on off switch, described as turning on a garbage disposal.1 Once cleaned out, the results were increased dopamine level and lower build up of alpha synuclein proteins.2 These results might benefit people with certain degenerative brain disorders.

Dosing of nilotinib may well be different for people with PD than for those with leukemia. The goal of most cancer drugs is to vigorously attack a disease target. The initial study used relatively low doses and still demonstrated improvement in motor and cognitive functions. The lower dose is being used to incite a reaction, not destroy everything in the path that the drug encounters. Studies will examine the long-term effects, safety, and tolerability of nilotinib in people with Parkinson’s disease. This includes dosing ranges to better understand the impact on long-term use. The goal is to verify and expand on previous nilotinib findings.

Hope or hype?

In addition to Georgetown, The Michael J. Fox Foundation for Parkinson’s Research, the Van Andel Research Center, and The Cure Parkinson’s Trust are all involved in multi-center Phase II trials.3 There are other instances of repurposing drugs that have proved beneficial in medical treatment. The involvement of all of these prestigious Parkinson’s organizations is evidence of the hope nilotinib is raising for clinical use as a possible treatment to slow the progression of or cure Parkinson’s disease.

  1. Hamilton, J. Cancer Drug That Might Slow Parkinson's, Alzheimer's Headed For Bigger Tests. Published March 15, 2017. Accessed online October 19, 2017.
  2. Kegel, M. Cancer Drug Raises Dopamine Levels, Eases Parkinson’s Symptoms in Early Study in Patients. Published July 18, 2016. Accessed online October 19, 2017.
  3. The Science of Parkinson’s disease. Nilotinib: The other phase II trial. Published July 31, 2017. Accessed online October 19, 2017.