Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a neurodegenerative disorder that affects the nerve cells responsible for controlling voluntary muscles. It is characterized by progressive degeneration and loss of motor neurons in the brain and spinal cord, leading to muscle weakness, paralysis, and respiratory failure.
ALS can strike anyone regardless of age, gender, or ethnicity, although it is more commonly diagnosed in individuals between 40 and 70. The exact cause of ALS is still unknown, and there is currently no cure.
A recent study published in the journal Cell Stem Cell suggests that Ropinirole, a medication commonly used to treat Parkinson’s disease, may also be effective in treating amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
Researchers from Japan conducted a clinical trial involving 20 individuals with ALS receiving care at Keio University Hospital. The participants did not have genetic predispositions to ALS and had lived with the disease for an average of 18 months.
The trial was conducted double-masked for the first 24 weeks, meaning neither the participants nor the doctors knew who received Ropinirole and who received a placebo—afterward, those who wanted to continue treatment received Ropinirole for 24 weeks. At the end of the trial, seven individuals who received Ropinirole and one who received the placebo completed the entire regimen.
The researchers assessed various measures throughout the trial and four weeks after the treatment ended. These measures included self-reported physical activity and independence in eating and drinking, activity data from wearable devices, and changes in mobility, muscle strength, and lung function as measured by physicians.
The results indicated that participants who received Ropinirole during both trial phases were more physically active than the placebo group. They also experienced slower rates of decline in mobility, muscle strength, and lung function and had higher survival rates.
Dr. Hideyuki Okano, the chair of the Department of Physiology at Keio University School of Medicine, explained that in a drug screening study using patient human induced pluripotent stem cells (iPS cells), Ropinirole exhibited several effects on ALS molecular pathology.
These effects included reducing antioxidant stress, protecting mitochondria, inhibiting the formation of abnormal protein aggregations (TDP-43 and FUS), inhibiting apoptosis (cell death), and inhibiting lipid peroxide. These multiple effects are believed to contribute to the amelioration of ALS symptoms.
The researchers also investigated the mechanisms behind Ropinirole’s effectiveness by growing motor neurons in individuals with and without ALS. They observed distinct differences in structure, gene expression, and metabolite concentration between the two groups.
Furthermore, those who took Ropinirole had less pronounced differences. The researchers were able to predict the effectiveness of Ropinirole treatment based on in vitro results.
While the study’s findings are promising, experts caution that it is a small and early study with few participants. Dr. Alessandro Di Rocco, a neurologist at Northwell Lenox Hill Hospital, emphasized the need for further research and noted that the study’s rationale, which involved screening potential drugs using induced pluripotent stem cell-derived motor neurons from ALS patients, could be a groundbreaking approach to drug development. However, more comprehensive studies are necessary to determine the drug’s efficacy and potential toxicity.
Overall, the study suggests that Ropinirole shows promise in delaying the progression of ALS and improving symptoms. Further research is needed to validate these findings and explore the drug’s potential as a treatment for ALS.