A former diabetes trial drug could offer new hope in the treatment of Parkinson’s disease, say scientists, whose recent trial findings indicated that the medication could improve motor symptoms linked to the disease.
The drug (NLX-112), 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 – holds promise that a new multi-faceted treatment could be in reach by 2030, said Parkinson’s UK.
The findings from the phase 2a trial of the drug, developed by the biopharma company Neurolixis, have shown promising results that were revealed at the World Parkinson’s Congress in Barcelona, Spain, last week, reports The Independent.
The trial found that there was significant reduction in movement symptoms, such as slowness, stiffness and tremor, in people taking the drug, which matches the performance of the best available medications 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.”
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 10 years, experts suggest.
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 released in March showed 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 – things like slowness, stiffness and tremor.
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 anyone 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.”
Twenty-six people living with Parkinson’s and dyskinesia were enrolled in the trial, and 22 of them completed the eight-week trial at several centres in Sweden.
Fifteen people received NLX-112 and seven received a placebo, all given 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.
Study details
Study to Assess the Safety, Tolerability and Preliminary Efficacy of NLX-112 Versus Placebo in L-dopa-induced Dyskinesia
Published on ClinicalTrials.gov on 20 March 2023
Brief Summary
This is a double-blind, randomised, placebo-controlled Phase 2a study evaluating the safety, tolerability, and preliminary efficacy of up to 2 mg/day (1 mg BID) of NLX-112 versus placebo in patients with moderate to severe L-DOPA induced dyskinesia (LID) in Parkinson's disease (PD). NLX-112 will be up-titrated to either 2 mg/day or to the highest well-tolerated dose less than 2 mg/day over 4 weeks, maintained at the well-tolerated dose for an additional 2 weeks, and then down-titrated over 2 weeks.
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