HQ Team
June 7, 2023: Ropinirole, a drug already approved for the treatment of Parkinson’s disease, has shown potential as a treatment for amyotrophic lateral sclerosis (ALS) also known as Lou Gehrig’s disease., according to a recent study published in the journal Cell Stem Cell.
Researchers at the Keio University Hospital in Japan conducted a clinical trial involving 20 ALS patients to investigate the efficacy of ropinirole.
The trial enrolled ALS patients who did not carry genetic predispositions for the disease and had an average disease duration of 18 months. The trial was double-blind for the initial 24 weeks, meaning both participants and doctors were unaware of who received ropinirole or a placebo. Afterwards, any participant willing to continue received ropinirole for the next 24 weeks.
Ultimately, seven participants received ropinirole throughout the trial, while one person completed the entire regimen with the placebo. The researchers assessed the participants for the entire duration of the trial and followed through for four weeks after completion. These measures included participants’ self-reported physical activity, their ability to eat and drink independently, wearable device data, and physician-measured changes in mobility, muscle strength, and lung function.
Results
The results indicated that participants who received ropinirole during both phases of the trial exhibited higher physical activity levels than the placebo group. They also experienced slower rates of decline in mobility, muscle strength, and lung function, and were more likely to survive.
Dr. Hideyuki Okano, the chair of the Department of Physiology at Keio University School of Medicine, explained that their drug screening study using patient human induced pluripotent stem cells (iPS cells) identified multiple effects of ropinirole on ALS molecular pathology. These effects included antioxidant stress, mitochondrial protection, inhibition of abnormal protein aggregation formation (specifically TDP-43 and FUS), inhibition of apoptosis, and inhibition of lipid peroxide. These multi-target effects are believed to contribute to the reduction of ALS symptoms.
Ropinirole
The researchers also explored the mechanisms underlying ropinirole’s effectiveness by studying motor neurons grown from ALS patients and those without the disease. They observed distinct differences in structure, gene expression, and metabolite concentration, with the ropinirole group showing less pronounced differences. Based on these in vitro results, the scientists were able to predict the efficacy of ropinirole treatment.
While the study’s findings are promising, experts say that it is a small and early study. Further research is necessary to validate these findings and establish the drug’s safety and efficacy for ALS patients
Ropinirole is an FDA-approved medication used for restless leg syndrome, as well as early and advanced Parkinson’s disease. It improves motor function and reduces motor decline in Parkinson’s patients. The drug can also alleviate sleep disturbances and nocturnal psychosis associated with Parkinson’s disease. Some common side effects of ropinirole include nausea, dizziness, and vomiting.
