Amyotrophic lateral sclerosis (ALS)also known as son of Lou Gehrig, is a fatal motor neuron disease that causes people to gradually lose control of their muscles. There is no cure, and current treatments focus on symptom relief and supportive care.
“ALS is totally incurable and is a very difficult disease to treatexplained senior author and physiologist Hideyuki Okano of Keio University School of Medicine in Tokyo. According to the research, the Parkinson’s drug ropinirole delayed disease progression by an average of 27.9 weeks.
The use of ropinirole
To test the safety and efficacy of ropinirole in patients with sporadic, i.e. non-familial amyotrophic lateral sclerosis (ALS), the research team recruited twenty patients being treated at Keio University Hospital in Japan. None of the patients carried disease-predisposing genes and, on average, they had been living with amyotrophic lateral sclerosis (ALS) for twenty months.
The study was conducted in double blind for the first twenty-four weeks, which means that patients and doctors did not know who was receiving ropinirole and who was receiving placebo. Then, for the next twenty-four weeks, all patients who wished to continue were knowingly given ropinirole. Many dropped out, partly due to the COVID-19 pandemic, and therefore only 7 of 13 ropinirole-treated patients and 1 of 7 placebo-treated patients were monitored for the full year. However, no patients withdrew for safety reasons.
Efficacy of the drug
To determine whether the drug was effective in slowing the progression of amyotrophic lateral sclerosis (ALS), the researchers monitored a number of different measures throughout the duration of the study and for four weeks after the end of treatment. These include changes in patients’ self-reported physical activity and in their ability to eat and drink independently, physical activity data from wearable devices, and changes in mobility, muscle strength, and lung function measured by doctor.
“We found out that ropinirole is safe and tolerable for ALS patients and shows therapeutic promise to help them support daily activity and muscle strength,” said first author Satoru Morimoto, a neurologist at Keio University School of Medicine in Tokyo.
Patients who received ropinirole during both phases of the study were more physically active than patients in the placebo group. They also showed slower rates of decline in mobility, muscle strength and lung function and were more likely to survive.
The benefits of ropinirole over placebo became increasingly apparent as the study progressed. However, patients in the placebo group who started taking ropinirole midway through the study did not experience these improvements, suggesting that treatment with ropinirole can be only useful if started earlier and administered for a longer duration.
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