Full results of repurposed cancer drug trial highlight potential benefits

Previous top line results indicated that, while safe, Nilotinib failed to show benefit. The full results now show that there may be some potential benefits.

Full results of the US-based NILO-PD clinical trial have been published in the research journal JAMA Neurology. They show that those taking nilotinib may have had some better outcomes and positive biological changes compared to the placebo group. 

But the findings are not clear cut and any positive effects will need confirming by larger trials.

The clinical trial

The NILO-PD phase 2 trial recruited 75 participants with moderate to advanced Parkinson's to take low doses of an approved cancer drug called nilotinib that is used to treat leukaemia.

Participants were randomly assigned to receive nilotinib at 150mg or 300mg, or a placebo daily over 12 months. Neither the researchers nor participants knew who was receiving what, this is called a double-blind trial.

The results

Top line results were released earlier this month in an effort to keep the Parkinson’s community up to date with the latest developments. While, these early results showed the low dose nilotinib was safe, no significant benefits were reported.

Now, full results suggest that those taking nilotinib may have performed better in certain measures of Parkinson’s, including motor testing and quality of life. Nilotinib was also seen to potentially reduce the levels of two toxic proteins associated with Parkinson’s, alpha synuclein and tau, and boost dopamine production.

However, the beneficial effects are not clear cut and no significant differences were seen in motor and nonmotor outcomes between the nilotinib groups and the placebo group. 

Dr Beckie Port, Research Communications Manager, comments:

“These full results add to our understanding of the potential of nilotinib for treating Parkinson’s but there are still unanswered questions. 

“Future trials of nilotinib or drugs that work in a similar way may demonstrate beneficial effects. But to give clinical trials the best chance of success, it is vital we invest in better, faster and more accurate ways to measure the symptoms of Parkinson’s and improved tools for use in clinical trials.”