A Parkinson’s drug has shown promise as a treatment for MND (motor neurone disease) and ALS (amyotrophic lateral sclerosis) in an early-stage clinical trial.
The findings have emerged from a double-blind trial conducted by Japanese researchers with a group of 20 participants – a number consistent with early-stage clinical tests – who were all MND patients without a genetic predisposition to the disease.
They were administered either the Parkinson’s treatment ropinirole hydrochloride or a placebo over the first 24 weeks of the trial.
Those who received the treatment were found to have their MND progression slowed by about 28 weeks on average.
as the clinical trial was conducted during the COVID-19 pandemic, several participants elected to exit the trial.
But based on the results, the researchers are confident the drug is safe for use as a treatment for motor neurone diseases like ALS. They will now progress to a stage three clinical trial.
“With this trial, we have shown that [ropinirole] is safe to use in ALS patients and that it potentially has some therapeutic effect, but to confirm its effectiveness we need more studies, and we are now planning a phase 3 trial for the near future,” says senior researcher Dr Hideyuki Okano, from Tokyo’s Keio University School of Medicine.
Cultivated stem cells from the study participants appears to show the mechanism by which ropinirole impedes MND’s advance. The researchers converted patient stem cells into motor neurones and found structural and genetic differences – including shorter axons and dendrites – when comparing them to healthy baselines.
Ropinirole appears to prevent these important connective projections from shortening, as well as suppressing around 29 genes associated with increase cholesterol synthesis in MND patients.
Motor neurone diseases see the body’s nervous system deteriorate over time as nerve cells die off, leading to muscle weakness and eventually paralysis. In Commonwealth countries like the UK and Australia it is usually referred to as MND, whereas the US typically describes the disease as ALS – the most common form of motor neurone degeneration – or occasionally Lou Gehrig’s disease after the baseballer who retired from the game after being diagnosed at the age of 36.
Parkinson’s disease is a neurodegenerative disease which diminishes a person’s ability to control their movements.
The trial measured self-reported physical activity levels, independent eating and drinking, activity data from wearable devices and clinically-monitored mobility, muscle strength and lung function changes.
But not everyone who received the drug drew benefit. Patients in the placebo group who commenced taking ropinirole after the first 24 weeks of the trial showed now significant improvement to their condition.
This suggests earlier interventions with the treatment would be favoured.