Breakthrough progress has been made in the study of amyotrophic lateral sclerosis (ALS). According to the official website of Umo University in Sweden on the 27th, a US developed antisense oligonucleotide (ASO) drug, Toferson, significantly slowed down the disease progression of a patient with ALS. After taking the medication for 4 years, the patient can still climb stairs, stand up from chairs, eat and speak normally, and live a positive and fulfilling social life. The patient comes from southern Sweden and suffers from ALS disease caused by SOD1 gene mutation. ALS, commonly known as ALS, is a chronic progressive neurological disorder that affects upper and lower motor neurons, as well as muscles in the trunk, limbs, and head and face. Since the summer of 2020, the patient has been participating in a phase III clinical study, receiving experimental treatment every 4 weeks. The aim of this study is to evaluate a novel gene therapy developed for patients with SOD1 mutated ALS. By intrathecal injection, targeting the mRNA of the mutated SOD1 gene promotes its degradation, thereby reducing the mutation type SOD1 protein and neurofilament light chain NFL (a biomarker of ALS). When diagnosed in 2020, the concentration of NFL in the patient's cerebrospinal fluid was as high as 11000 nanograms per liter. After 4 years of treatment, the concentration of NFL decreased to 1200-1290 nanograms per liter, a decrease of nearly 90%. Scientists use a scale for measuring the functional level of a patient's body, with a healthy individual score of 48. In the past 18 months, the patient's functional level has remained at 35-37 points. The research team pointed out that generally speaking, patients with this type of ALS lose 1-1.5 points in their physical function level per month. If left untreated, the disease can rapidly worsen and lead to severe disability within 6-12 months. But this patient can still climb stairs after 4 years of illness, which is a miracle to some extent. Research leader and neurologist Peter Anderson from Umeo University pointed out that ALS has multiple types, with only 2-6% of patients being caused by mutations in the SOD1 gene. It is currently unclear whether this drug is effective for other types of ALS. Moreover, not all experimental participants were able to achieve the same positive results, which may be related to dosage or when treatment began. They hope to further study to clarify these issues.
Edit：GuoGuo Responsible editor：FangZhiYou
Source：People.cn
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