United Nations drug used against Parkinson’s is safe to treat patients with amyotrophic lateral sclerosis (ALS) and delayed disease progression for an average of 27.9 weeks in a small clinical trial, although further testing is needed to confirm its efficacy.

ALS is a deadly motor neuron disease that causes people to gradually lose control of their muscles. There is no cure and current treatments focus on reducing symptoms and providing supportive care.

A Japanese study published today by Cell Stem Cell has tested the drug ropinirole in a clinical trial with patients with sporadic ALS, that is, non-familial. Lead author Hideyuki Okano of Tokyo Keio University said this trial has shown that the drug is safe in ALS and that it «potentially has some therapeutic effect, but to confirm its efficacy» more studies are needed, so it is planned a phase 3 trial «for the near future.»

The trial enrolled 20 patients with ALS who did not carry genes that predispose to the disease and, on average, had been diagnosed for 20 months. Some people responded better to the drug than others, and although the reasons are not clear, the researchers believe that it is probably due to genetic differences that they hope to identify in future studies.

The trial was double-blind for the first 24 weeks, meaning that neither the patients nor the doctors knew who received ropinirole and who received placebo. For another 24 weeks the drug was administered to all patients who wished to continue.

Maintain daily activity

Many dropped out of the trial, partly because of the covid pandemic, so only 7 of 13 patients treated with ropinirole and 1 of 7 with placebo followed by the drug were monitored throughout the year. The team highlighted that no patient had dropped out of the clinical trial for reasons of drug safety.

To determine if the drug was effective in slowing the progression of ALS, a series of measures were monitored during the trial and four weeks after the end of treatment. The team found that ropinirole «shows therapeutic promise» in ALS patients to help maintain daily activity and muscle strength,» said Satoru Morimoto, a co-author of the study.

Patients who received ropinirole during both phases of the trial were more physically active than those in the placebo group and showed slower rates of decline in mobility, muscle strength and lung function, and were more likely to survive. The benefits of ropinirole relative to placebo became increasingly pronounced as the trial progressed.

As for people in the placebo group who were able to take the drug halfway through the trial, they did not experience these improvements, «suggesting that ropinirole treatment may only be useful if it is started earlier and is administered for a longer duration,» he explains. publication in a statement.

Mother cells

The researchers studied the mechanisms underlying the effects of ropinirole and looked for molecular markers of the disease. To do this, they generated induced pluripotent stem cells from the patients’ blood and transformed them into motor neurons in the laboratory. «We found a very striking connection between a patient’s clinical response and their motor neuron response in vitro,» Morimoto said.

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Patients whose motoneurons responded robustly to ropinirole «in vitro» had much slower clinical disease progression with treatment, whereas those who responded suboptimally showed faster disease progression despite taking the drug. .

This method of culturing and assaying motor neurons from patient-derived induced pltipotent stem cells could be used clinically to predict drug efficacy in a given patient, the team suggests.