New Gene Therapy Offers Hope for 'ALS' Patients

Breakthrough: Antisense Oligonucleotide Drug Toferson Significantly Slows ALS Patients' Disease Course

Amyotrophic lateral sclerosis (ALS) is a fatal disease that progressively paralyzes a patient's muscles, eventually leading to death. There is currently no cure, but in recent years, significant advances have been made in the field of gene therapy, offering new hope to patients.

An ALS patient with a mutation in the SOD1 gene experienced significant remission after treatment with Toferson

A patient from southern Sweden, who suffers from ALS due to a mutation in the SOD1 gene, has been participating in a Phase III clinical study since the summer of 2020, receiving treatment every four weeks with Toferson, an antisense oligodeoxynucleotide (ASO) drug administered via intrathecal injection, which targets the mRNA of the mutant SOD1 gene and promotes its degradation, thereby reducing the production of mutant SOD1 protein and neurofilament light chain NFL.

Prior to treatment, the patient had NFL concentrations in cerebrospinal fluid as high as 11,000 ng/l. After 4 years of treatment, the NFL concentration dropped to 1,200-1,290 ng/liter, a nearly 90% decrease. At the same time, the patient's level of functioning improved significantly. The level of functioning in healthy individuals, as assessed by the ALS scale, is 48 points, while the patient has maintained a score of 35-37 points for the past 18 months, which is rare among ALS patients.

opens a new direction for ALS treatment, but further research is needed

This study suggests that Toferson is effective in treating ALS caused by mutations in the SOD1 gene. However, the researchers noted that it is unclear whether the drug is effective for other types of ALS. In addition, not all participants in the trial achieved the same positive results, which may be related to the dose or when the treatment was started. They hope further research will clarify these issues.

Looking Ahead: Gene Therapy Offers Hope for ALS Patients, but Continued Exploration Is Needed

Toferson's success story opens up new directions for ALS treatment and offers lessons for gene therapy for other difficult-to-treat diseases. However, gene therapy technology is still in the developmental stage, and more research is needed to explore its scope of application and safety in the future. It is believed that with the advancement of technology, gene therapy will bring benefits to more patients.