Ropinirole Can Help Mitigate ALS Symptoms

Monday - June 26 2023, 17:56 UTC - 1 year ago

Keio University School of Medicine in Tokyo, Japan recently demonstrated that the drug ropinirole is safe for ALS patients and delayed the progression of the disease by an average of 27.9 weeks. The trial found ropinirole to be safe and tolerable for ALS patients and shows therapeutic promise at helping with daily activity and muscle strength, while also providing increased quality of life. These findings hold promise for developing more effective treatments to help slow the progression of ALS.

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease is a lethal neurological disorder that progressively results in individuals losing muscle control. Currently, there is no cure, and the primary focus of treatments is to alleviate symptoms and offer supportive care. Early clinical trial findings recently published in the journal Cell Stem Cell, demonstrated that the drug ropinirole, typically used for Parkinson’s disease, is safe for ALS patients and delayed the progression of the disease by an average of 27.9 weeks.

Some patients showed a greater response to the ropinirole treatment than others. Interestingly, the scientists were able to forecast this clinical responsiveness in a lab setting, by utilizing motor neurons derived from the stem cells of the patients.

"ALS is totally incurable, and it’s a very difficult disease to treat," says senior author and physiologist Hideyuki Okano of the Keio University School of Medicine in Tokyo. "We previously identified ropinirole as a potential anti-ALS drug in vitro by iPSC drug discovery, and with this trial, we have shown that it 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." .

To test ropinirole’s safety and effectiveness in patients with sporadic (i.e., non-familial) ALS, the team recruited 20 patients receiving care at Keio University Hospital in Japan. None of the patients carried genes predisposing to the disease, and, on average, they had been living with ALS for 20 months.

The trial was double-blinded for the first 24 weeks, meaning that the patients and doctors did not know which patients were receiving ropinirole and which were receiving a placebo. Then, for the following 24 weeks, all patients who wished to continue were knowingly administered ropinirole. Many patients dropped out along the way—partially due to the COVID-19 pandemic—so only 7/13 ropinirole-treated and 1/7 placebo-followed-by-ropinirole-treated patients were monitored for the full year. However, no patients dropped out due to safety reasons.

To determine whether the drug was effective at slowing the progression of ALS, the team monitored a variety of different measures throughout the trial and for 4 weeks after treatment concluded. These included changes in the patients’ self-reported physical activity and ability to eat and drink independently, activity data from wearable devices, and physician-measured changes in mobility, muscle strength, and lung function.

"We found that ropinirole is safe and tolerable for ALS patients and shows therapeutic promise at helping them sustain daily activity and muscle strength," says first author Satoru Morimoto, a neurologist at the Keio University School of Medicine in Tokyo.

Patients who received ropinirole during both phases of the trial 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 they were more likely to survive. The benefits of ropinirole relative to the placebo were most impressive when looking at providing more daily activity that could result in increased quality of life.

These findings are very exciting and important; although more research needs to be done, this trial holds promise to develop more effective treatments to help slow the progression of ALS. While ropinirole was not effective for all patients, for some, this drug was able to forestall the development of their disease for nearly a year.