ALS: Parkinson's drug ropinirole may be an effective treatment

• Ropinirole is a medication already approved as a treatment for Parkinson’s disease.
• Researchers are now saying the drug may be an effective treatment for amyotrophic lateral sclerosis (ALS).
• The Japanese scientists reached that conclusion after conducting a clinical trial involving 20 people being treated for ALS.

Ropinirole, a drug used to treat Parkinson’s disease, showed promise in delaying the progression of amyotrophic lateral sclerosis (ALS), according to a study published today in the journal Cell Stem Cell.

Researchers from Japan reached that conclusion after conducting a clinical trial to examine whether ropinirole could help people with ALS, also known as Lou Gehrig’s disease.

The researchers reported on 20 people receiving care at Keio University Hospital in Japan enrolled in the study. None of the participants carried genes that predisposed them to the condition. On average, they had lived with ALS for 18 months.

The trial was double-blind for the first 24 weeks, meaning the participants and doctors were unaware of who received ropinirole and who was getting a placebo. For the next 24 weeks, any participant who wanted to continue received ropinirole.

In the end, seven people receiving ropinirole and one person given placebo completed the entire trial regimen.

Results from the ALS drug clinical trial

The researchers looked at several measures throughout the trial and for four weeks after treatment concluded.

The measurements included:

• Participants’ self-reported physical activity and ability to eat and drink independently.
• Activity data from wearable devices.
• Physician-measured changes in mobility, muscle strength, and lung function.

The researchers reported that participants who received ropinirole during both phases were more physically active than the placebo group. They also had slower rates of decline in mobility, muscle strength, and lung function and were more likely to survive.

Dr. Hideyuki Okano, a professor and the chair of the Department of Physiology at Keio University School of Medicine, told Medical News Today that in their drug screening study using patient human induced pluripotent stem cells (iPS cells), they identified the following effects of ropinirole on ALS molecular pathology:

• antioxidant stress
• mitochondrial protection
• inhibition of abnormal protein (TDP-43 and FUS) aggregation formation
• inhibition of apoptosis
• inhibition of lipid peroxide

“These multi-target effects are thought to play a part in the mechanism of ALS symptom amelioration,” Okano said.

Why ropinirole might be effective in treating ALS

The researchers also investigated the mechanisms behind ropinirole’s effectiveness.

To do this, they grew motor neurons from people with ALS and those without the disease.

The researchers reported distinct differences in structure, gene expression, and metabolite concentration. The people who took ropinirole also had less pronounced differences.

The scientists said that they could predict the effectiveness of the ropinirole treatment based on the in vitro results.

“When iPS cells were established from all patients participating in the clinical trial and induced to differentiate into motor neurons, it was found that patient-derived motor neurons had significantly more fragile neurites,” Okano explained. “Treatment with ropinirole improved neurite fragility. However, we found that the motor neurons of the participants were stratified according to the degree of improvement of neurite fragility by ropinirole. Therefore, when patients in clinical trials were classified according to the responsiveness of iPS cell-derived motor neurons to ropinirole, it was found that the group of patients with well improved own motor neurons responded better to ropinirole clinically, and the progression of ALS was reduced.”

Reaction to the ALS drug study

“Despite the exciting results, this is a small, early study,” Dr. Alessandro Di Rocco, a neurologist at Northwell Lenox Hill Hospital in New York, told Medical News Today. “This is a very preliminary small clinical study with only a few patients continuing into the observation study who appeared to have a beneficial effect from the drug.”

“The study’s rationale is based on a novel research approach to screening potentially effective drugs, using ‘Induced pluripotent stem cell (iPSC)- derived motor neurons’ from ALS patients. With this method, it may be possible to test whether specific drugs can be directly effective in the affected organs and possibly accelerate the development of candidate drugs,” he added.

“Preliminary laboratory work is essential in determining potential efficacy and potential toxicity,” Di Rocco said. “In this case, safety and toxicity are less critical, as the drug is approved for different indications. The model of Induced pluripotent stem cell (iPSC)- derived motor neurons as in-vitro screening is potentially a ground-breaking approach to drug development.”

What you should know about ropinirole

Ropinirole is a medication approved by the U.S. Food and Drug Administration for restless leg syndrome as well as early and advanced Parkinson’s disease.

The drug improves “on” time (time of good motor function) and minimizes the “off” time (severe motor decline) of Parkinson’s.

It can also help with sleep disturbances and nocturnal psychosis, both associated with Parkinson’s disease.

Some side effects of ropinirole include can include nausea, dizziness, and vomiting.

Experts say you should contact your doctor if side effects do not disappear within a few weeks.

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