'Very encouraging' trial offers hope of a breakthrough in treatment for Parkinson's disease

A former diabetes trial drug could be used to treat Parkinson's disease within years, research suggests.

The drug, which was designed to treat dyskinesia - involuntary movements that are a common side effect in Parkinson's patients who take levodopa-based medication for several years - could also improve motor symptoms linked to the condition.

According to Parkinson's UK, the drug - NLX-112 - offers hope that a new multi-faceted treatment could be in reach by 2030.

The findings from a phase 2a trial of the drug, developed by the biopharma company Neurolixis, have shown promising results, which are being revealed at the World Parkinson's Congress in Barcelona, Spain.

In people taking the drug, there was significant reduction in movement symptoms, such as slowness, stiffness and tremor, the trial found.

The medication matches the performance of the best available drugs for two symptoms at low doses, experts say.

Adrian Newman-Tancredi, co-founder, president and chief executive of Neurolixis, said: "The positive effect seen on both dyskinesia and movement symptoms could make NLX-112 an important, dual efficacy therapy, thanks to its novel neurochemical mechanism, distinct to that of current medications for Parkinson's.

"The fact that such robust results were observed in this relatively small and short term study is very encouraging.

"We now need to carry out larger, longer studies as quickly as possible. If things go well, NLX-112 could be available by 2030."

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Which drugs are currently available?

Most current drug treatments for Parkinson's work by boosting or mimicking the effects of a neurochemical called dopamine inside the brain.

These therapies, notably levodopa, have been considered the gold standard since the 1960s and are effective, particularly in the early stages of the condition.

However, they become less effective over time and up to 50% of all people with Parkinson's develop dyskinesia within five years. Up to 80% experience it after ten years, experts suggest.

What is NLX-112?

NLX-112 works through a different mechanism to current Parkinson's therapies, and instead works through a serotonergic pathway which previous research has shown to be important in controlling movement.

Early results from the phase 2a trial, which was released in March, demonstrated that the drug had clear anti-dyskinesia effects that increased over the period of treatment.

Further analysis of the initial results suggests the drug also had a positive effect on the symptoms of Parkinson's.

Those enrolled in the trial showed a significant reduction in movement symptoms, such as slowness, stiffness and tremor.

These findings indicate that drugs targeting the serotonin system could provide important new treatments for managing movement symptoms in Parkinson's.

Dr Arthur Roach, director of the Parkinson's Virtual Biotech, at Parkinson's UK, said: "These unexpected benefits of NLX-112 are interesting for us and any one affected by Parkinson's.

"By targeting the serotonergic pathway to treat levodopa induced dyskinesias, NLX-112 could be pioneering a new treatment strategy for both dyskinesia and wider motor symptoms that would offer a different option for people to manage their Parkinson's.

"Parkinson's UK, in partnership with the Michael J Fox Foundation, has been on this journey with Neurolixis from the very early days, and these additional findings are testament to the potential that we saw in this approach.

"Our Parkinson's Virtual Biotech programme is fast-tracking work that could deliver treatments in years, not decades and successes like this reinforce why we're taking this bold approach."

How did the trial work?

Twenty-six people living with Parkinson's and dyskinesia were enrolled in the trial, and 22 participants completed the eight-week trial at several centres in Sweden.

Fifteen people received NLX-112 and seven received a dummy pill (or placebo).

People either received NLX-112 or the placebo in increasing doses during the initial four weeks, to minimise the potential side effects.

They stayed on the maximum dose for two weeks and then were weaned off the drug over another fortnight.

As well as finding that those who took the drug had improvements in the levodopa-induced dyskinesia and Parkinson's symptoms, the trial also found the drug was well tolerated and safe.

Katharina Klapper, director of clinical research at the Michael J Fox Foundation, said: "We're energised by the promising early results presented by the Neurolixis team to help treat levodopa-induced dyskinesia, which can be challenging and have a significant impact on patient’s lives and their ability to perform daily tasks.

"We are grateful to the study participants and the collaboration of the Parkinson's Virtual Biotech building on our shared goal of unlocking more opportunities in today's clinical research pipeline and better treatments for people and families."

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