Experimental drug treatment tofersen may be useful for treating inherited form of ALS

New research highlights the results of phase 1/2 clinical trial involving the experimental drug treatment tofersen.

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A study in the New England Journal of Medicine highlights the results of phase 1/2 clinical trial involving the experimental drug treatment tofersen, also known as BIIB067.

According to researchers at the Washington University School of Medicine, tofersen may be effective at reducing the severity of the inherited form of amyotrophic lateral sclerosis (ALS). The progressive neurodegenerative disease is caused by a mutation in the gene SOD1.

“Tofersen is an antisense oligonucleotide that mediates the degradation of superoxide dismutase 1 (SOD1) messenger RNA to reduce SOD1 protein synthesis. Intrathecal administration of tofersen is being studied for the treatment of amyotrophic lateral sclerosis (ALS) due to SOD1 mutations,” the findings read.

In the study, 50 participants received doses of either tofersen or placebo, administered for 12 weeks.

“A total of 50 participants underwent randomization and were included in the analyses; 48 participants received all five planned doses. Lumbar puncture–related adverse events were observed in most participants,” the co-authors stated in the findings.

“In adults with ALS due to SOD1 mutations, CSF SOD1 concentrations decreased at the highest concentration of tofersen administered intrathecally over a period of 12 weeks. CSF pleocytosis occurred in some participants receiving tofersen. Lumbar puncture–related adverse events were observed in most participants.”

The findings provide a glimpse of what is a potential drug treatment efficient at combating the effects of ALS, however, more research is needed.

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