In the United States alone, there are an estimated 20,000 people living with amyotrophic lateral sclerosis (ALS), also called as Lou Gehrig's disease. The fatal disease destroys the nerve cells which control breathing, walking, and eating. Very few people survive more than three years after being diagnosed.

Recent research led by the Washington University School of Medicine in St. Louis revealed that an investigational therapy for ALS would extend patient survival and reverses the signs of neuromuscular damage. The findings were published in The Journal of Clinical Investigation,  and have led to a phase one/two clinical trials in order to explore whether the drug could have a positive effect in patients with ALS caused by the SOD1 gene mutation.

Timothy Miller, MD, Ph.D., the David Clayson Professor of Neurology from Washington University said, "This drug had an impressive effect in mice and rats with just one or two doses; We don't know yet if this works in people, but we're very hopeful. We've completed the first phase of safety testing, and now we're working on finding the right dose."

Timothy Miller and his colleagues, in collaboration with Ionis Pharmaceuticals, tested DNA-based compounds such as antisense oligonucleotides, or oligos which block the body from making SOD1 protein.

Timothy Miller stated, "The phase one/two trial is really still a safety trial; There are not enough patients in it to really be able to accurately see an effect on disease. But we're on the cusp of testing the hypothesis that people with ALS caused by mutations in SOD1 can benefit from this treatment. We predict the effect will be good, but we can't know until we test it."