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Hope for a cure for small group of ALS patients

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Significant progress has been made in the search for a cure for: Lettucethe amyotrophic lateral sclerosis neurodegenerative disease that causes a degeneration of motor neurons, the nerve cells of the brain and spinal cord that allow movement of the skeletal muscles. A study just published in the New England Journal of Medicine showed thatn improvement of biomarkers of disease in treated patients tofersen, an antisense oligonucleotide (ASO) that selectively acts on messenger RNA and blocks the synthesis of the altered protein. The Molinette Città della Salute hospital in Turin also took part in the international study.

Therapy only affects 1% of ALS patients

It’s good to clarify that it’s not about universal therapy against ALS why the treatment works in patients who carry the Sod1 gene mutation and therefore only interests a small percentage of people with ALS. “The publication of the study is exceptional news and the work is widely known in the scientific community, but it is important to emphasize that only a very small proportion of patients are involved,” he explains. Nile coast, neurologist and researcher at the IRCSS San Raffaele Hospital in Milan -. To understand, 10% of ALS cases have known (other cases in the family) and within this 10% only 10-20% of patients have the Sod1 mutation, so about we may not reach 1% of the population with amyotrophic lateral sclerosis, in Italy several dozen patients. This does not detract from the value of the discovery that has affected something hitherto impenetrable ».

I study

The involved experimental research 108 people affected by ALS with mutation in the Sod1 gene (72 participants received lumbar injection tofers and 36 received placebo). The volunteers were divided into patients with rapid and slow progression and the study had two phases: a first 6 months and a second extension of the study which was still ongoing. Relevant, according to what Turin emphasizes, is the significant reduction of the Sod1 protein and neurofilaments, typical markers of the disease, in cases subjected to this innovative therapy. “The positive effect of the drug is clearly manifested during the first year of treatment and then persists over time,” said the professor Adriano Chicdirector of the regional center of expertise for ALS at the Molinette Hospital of the City of Health in Turin, one of the authors of the work.

No clinical improvement for the time being

The researchers emphasize in the conclusions, in addition to the serious neurological effects in 7% of the patients who received the drug, the fact that there was no clinical improvement in patients. At least not yet «Because the disease is devastating – adds Nilo Riva – from which there is no turning back, the researchers they saw no improvement in the patients’ condition or a clinical benefit, but only an improvement in the markers, i.e. the reduction in the concentration of Sod1 in the spinal fluid and a decrease in the light chains of the neurofilaments, two markers of the disease. Although the result is exceptional, no tangible benefit to the patient has yet been seen, given the small number of people treated and the still limited time elapsed since treatment.”

Source: Corriere

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