Results of phase 2 nilotinib study show no benefit

Top-line results from the US-based phase 2 clinical trial of nilotinib show that, while safe and tolerable, it did not have beneficial effects.

The NILO-PD phase 2 trial recruited 75 participants with moderate to advanced Parkinson's to take an approved cancer drug called nilotinib.

Participants were randomly assigned to receive nilotinib or a placebo daily over 12 months with neither the researchers nor participants knowing who was receiving what.

Now, having unblinded the study, the researchers have released early top-line results that suggest the drug does not benefit those with Parkinson's.

What is nilotinib?

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

There is interest in repurposing the drug for Parkinson’s as it reduces the activity of c-Abl, a protein linked to a number of cellular pathways that are affected in the condition.

By finding drugs with hidden potential for Parkinson's and rapidly moving them into clinical trials. The idea is that repurposing approved medicines can make new drugs available for people with Parkinson's much more quickly, easily and cheaply.

Trials of nilotinib in Parkinson's

In 2015, the results of a phase I study of nilotinib made media headlines. The study found that people with Parkinson’s who took low doses of the repurposed drug over a period of 24 weeks saw improvements in thinking, movement and non-motor symptoms. 

Based on these results, a phase 2 trial was launched in 2017 to further investigate the safety and potential benefits of nilotinib for treating Parkinson's. 

Dr Beckie Port, Research Communications Manager, comments:

“The top-level findings were released early in a commendable effort to keep the Parkinson’s community informed of new scientific findings as quickly as possible. It is likely more information will be available when the full results have been analysed.

“There is much still to learn about the potential of drugs that work in the same way as nilotinib. While this study has not produced positive findings, drugs that target the c-Abl protein continue to hold promise.”

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Read more about this study and discover the latest progress in Parkinson's research with our Research Blog.