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Study of Treatment for Genetic ALS

An experimental treatment is showing promise for treating the genetic form of amyotrophic lateral sclerosis, CNN reports.

Researchers led by Washington University in St. Louis' Timothy Miller treated ALS patients harboring mutations in their SOD1 gene — mutations in that gene cause between 15 and 20 percent of familial ALS — with the antisense oligonucleotide drug tofersen, it adds. The 50 patients in the study were randomized to receive 20 milligrams, 40 milligrams, 60 milligrams, or 100 milligrams of tofersen or a placebo for 12 weeks. After that time, CNN reports, the researchers found tofersen treatment led to a reduction in muscular decline as well as to a reduction in SOD1 protein in patients' spinal fluid.

"Reducing the level of the SOD1 protein would be a good thing for people with SOD1 mutations causing ALS," Miller tells CNN. "The SOD1 protein with the mutation is what's causing the toxicity. It's what's causing ALS in people with a genetic change in SOD1."

The researchers are to present these results at the upcoming of American Academy of Neurology annual meeting, it adds.