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Phase II clinical trial of cancer drug announced

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US researchers have announced the launch of a Phase II clinical trial of the cancer drug nilotinib in people with Parkinson's.

The study, based at Georgetown University Medical Center in Washington DC, US, will investigate the drug’s safety and its potential for treating Parkinson’s symptoms. 

It follows on from encouraging results of a small phase I trial of the drug.

What do we know about nilotinib so far?

Nilotinib is a drug that is already approved for treating leukaemia.

In 2015, results of a phase I study suggested that people with Parkinson’s who took nilotinib over a period of 24 weeks saw improvements in thinking, movement and non-motor symptoms.

Although the initial phase I study of nilotinib showed promising results, its small size, and the lack of a placebo group left many questions unanswered.

However, the trial was small, involving only 12 patients, and didn’t include a placebo group to compare the drug against.

The results of the trial were published in the Journal of Parkinson's Disease.

About the trial

This 12 month trial is set to involve 75 people with moderate Parkinson’s.

Participants will be randomly assigned to receive nilotinib or the placebo and neither researchers nor participants will know which treatment they are getting.

The research team will evaluate the safety of low doses of the drug and monitor its effect on Parkinson’s symptoms and biomarkers in cerebrospinal fluid - the colourless fluid that surrounds the brain and spinal cord. They hope this will determine whether nilotinib is safe and its promise as a future treatment.

Participants will continue to take nilotinib and be monitored for a further 12 months to understand the long-term effects of the drug.

Further details about the study can be found on the clinicaltrials.gov website.

The next step for nilotinib

Sophie McLachlan, Research Communications Officer, comments:

"Repurposing drugs involves finding treatments like nilotinib – that are already approved and in use for other conditions – that might have hidden benefits for Parkinson’s. It’s hoped this could make new treatments available much more easily, quickly and cheaply.

"Although the initial phase I study of nilotinib showed promising results, its small size, and the lack of a placebo group left many questions unanswered.

"This larger controlled trial is a vital step towards improving our understanding of whether this drug could be a safe and effective treatment for Parkinson’s. We’ll be keeping a close eye on progress and reporting any developments."