Nilotinib (Tasigna) for Parkinsons: Questions and Answers

Nilotinib (Tasigna) for Parkinsons: Questions and Answers.

What is nilotinib (Tasigna)?

Nilotinib is a FDA approved cancer drug for approved for use in certain types of chronic leukemia. Nilotinib is a protein kinase inhibitor that works by blocking abnormal protein signals which tell cancer cells to divide and multiply. The drug thus helps slow the growth and spread of cancer cells. It is not approved for use in Parkinson’s disease.

Why all the excitement about nilotinib in Parkinson’s disease?

Over the last several years a handful of research studies in cell and animal models of Parkinsonism have demonstrated positive effects with nilotinib (Hebron et al, Hum Mol Genet 2013; Karuppagounder et al, Sci Rep 2014; Mahul-Mellier et al, Hum Mol Genet 2014). Parkinson’s disease is characterized pathologically by the loss of dopamine neurons in the substantia nigra (brainstem) and abnormal accumulation of the protein alpha-synuclein in the brain. As a protein kinase inhibitor, nilotinib appears to decrease the activity of a certain protein, Abl, and signals cells to clear alpha-synuclein. In animal models nilotinib not only reduced alpha-synuclein but also another toxic protein called tau, and rescued dopamine cell loss and behavioral deficits.  These findings were exciting and led to a recent patient study with nilotinib.

Recently it was announced that researchers at Georgetown University performed a Phase I study to test the safety and tolerability of nilotinib treatment in patients with Parkinson’s disease. This study was small, involving only about a dozen patients, and was open-label, meaning the patients were not blinded and knew that they were getting the drug, nilotinib. The preliminary results were just announced and demonstrate that the drug at doses used was safe and tolerable. Several of the patients on drug additionally reported remarkable improvement in their clinical symptoms. While these results are significant and certainly exciting, it is important to note that this study was not designed to show efficacy of the drug, only safety and tolerability. The findings have not been published yet either. Future Phase II studies will be needed to determine whether nilotinib improves symptoms in Parkinson’s disease. These studies will need to include a larger number of patients, multiple sites, blinded rating, and a placebo-drug group for comparison.

Should I take nilotinib?

In short, no, unless you take part in a clinical trial.While an exciting development for Parkinson’s disease and possibly other neurodegenerative disorders, more studies will be needed to clearly establish nilotinib as a safe and effective drug for these disorders. Other similar candidate drugs are also under investigation and it may be worthwhile to wait for these also to be tested to make sure the right drug is used. Also important, nilotinib is a chemotherapeutic drug and not without potential side effects. Nilotinib can cause an arrhythmia (abnormal heart rhythm that may cause you to faint, seize, or sudden death), stroke, abnormal bleeding/hemorrhage, liver disease, pancreatitis, pneumonia, and thyroid issues. There are also many other more common side effects.

How can I participate in a clinical trial for nilotinib?

For now, please contact the Georgetown University National Parkinson Foundation Center of Excellence ({}) and follow the website for announcement of new trials.

Dr. Okun posted on behalf of the National Parkinson Foundation on this issue and you can read that on the NPF website.

About the Author


Nikolaus McFarland

Dr. Nikolaus McFarland is a movement disorders expert with special interest in atypical parkinsonisms/Parkinson-plus disorders. He directs the PSP/Atypical Parkinson's clinical-research program here at UF.…

Read all articles by Nikolaus McFarland