2023-06-03 08:07:06
In Japan, an early clinical trial on 20 patients with Charcot’s disease, incurable to date, offered promising results. Elements of explanation on the tests carried out and their results.
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Amyotrophic lateral sclerosis (ALS), also called Charcot’s disease, is a neurodegenerative disease that destroys motor neurons (or motoneurons) and leads to progressive paralysis of the muscles involved in voluntary motricity (for walking, speaking or even swallowing). There are currently three drugs approved by the Food and Drug Administration for this incurable disease but their benefits remain limited, focusing mainly on the reduction of symptoms.
Delay disease progression
For Parkinson’s disease, ropinirole is an effective drug that overcomes the dopamine deficiency – characteristic of the disease – and in particular improves walking time. Researchers are interested in this treatment already approved to treat other neurodegenerative diseases such as ALS. The results of an early clinical trial, published in the journal Cell Stem, show that ropinirole is promising against ALS, delaying its progression by an average of 27.9 weeks. ” We found that ropinirole is safe and tolerable for patients with ALS and shows therapeutic promise in helping them maintain daily activity and muscle strength. », said co-author Satoru Morimoto, a neurologist at Keio University School of Medicine in Tokyo.
The research team recruited 20 Japanese patients with non-familial ALS (none of the patients carried genes predisposing to the disease). For the first 24 weeks of the trial, these people received either ropinirole or a placebo, without knowing what they were getting. While some patients had to drop out of the trial because of the Covid-19 pandemic, the others all received the drug afterwards.
The mobility, muscle strength and lung function of patients who had received ropinirole during the two phases of the trial decreased less rapidly than those of patients who had received a placebo in the first phase. For greater effectiveness, the treatment must therefore be administered over a longer period.
Patients with variable responses
The researchers also looked for molecular markers of the disease, in order to elucidate the mechanisms underlying the effects of ropinirole. From the patients’ blood, they generated induced pluripotent stem cells and cultured them in the laboratory to differentiate them into motor neurons. They found that motor neurons from ALS patients showed differences in structure and gene expression compared to healthy motor neurons, but that ropinirole reduced these differences.
« We found a very striking correlation between a patient’s clinical response and the response of his motor neurons in vitrexplains the neurologist. Patients whose motor neurons responded robustly to ropinirole in vitro had significantly slower clinical disease progression with treatment, whereas patients with suboptimal response had significantly more rapid disease progression despite this same treatment. »
It’s unclear exactly why some patients respond better to ropinirole than others, but the researchers hypothesize genetic differences that they hope to reveal in future studies.
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