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Parkinson’s disease drug ropinirole safely slowed progression of ALS in clinical trial

  • Ropinirole is a drug already approved for the treatment of Parkinson’s disease.
  • Researchers now say the drug could be an effective treatment for amyotrophic lateral sclerosis (ALS).
  • The Japanese scientists came to this conclusion after conducting a clinical trial involving 20 people being treated for ALS.

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

Japanese researchers came to this conclusion after conducting a clinical trial to determine whether ropinirole may help people with ALS, also known as Lou Gehrig’s disease.

Researchers reported 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 disease. On average, they had been living with ALS for 18 months.

The trial was double-blind for the first 24 weeks, meaning participants and doctors did not know who was getting ropinirole and who was getting a placebo. For the next 24 weeks, any participant wishing to continue received ropinirole.

Ultimately, seven people receiving ropinirole and one person receiving a placebo completed the entire trial regimen.

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

The measures included:

  • Participants’ self-reported physical activity and the ability to eat and drink independently.
  • Wearable device activity data.
  • Doctor-measured changes in mobility, muscle strength, and lung function.

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 Okanoprofessor and head of the department of physiology at Keio University Medical School, said Medical News Today that in their drug screening study using human induced pluripotent stem cells (iPS cells) from patients, they identified the following effects of ropinirole on the molecular pathology of ALS:

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

“These multi-target effects are thought to play a role in the mechanism of improving ALS symptoms,” Okano said.

The researchers also studied the mechanisms behind the effectiveness of ropinirole.

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

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

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

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

“Despite the exciting results, this is a small, early study,” Dr Alessandro Di Roccoa neurologist at Northwell Lenox Hill Hospital in New York, said Medical News Today. “This is a very preliminary small clinical study with only a few patients continuing the observational study who appeared to benefit from the drug.”

“The rationale for the study is based on a new research approach for screening potentially effective drugs, using “motor neurons derived from induced pluripotent stem cells (iPSCs)” from ALS patients. With this method, it may be possible to test whether specific drugs can be directly effective in affected organs and possibly accelerate the development of drug candidates,” he added.

“Preliminary laboratory work is essential to determine potential efficacy and potential toxicity,” Di Rocco said. “In this case, safety and toxicity are less critical because the drug is approved for different indications. Modeling induced pluripotent stem cell (iPSC)-derived motor neurons as an in vitro screen is potentially a breakthrough approach for drug development.

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

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

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

Some Side Effects of Ropinirole include may include nausea, dizziness and vomiting.

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


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