School of Medicine

New ALS therapy in clinical trials

Robert Bucelli, MD, PhD, measures Gregory Easter's neuromuscular function. Easter, who has an inherited form of amytrophic lateral sclerosis, a fatal neuromuscular disease, is taking part in a clinical trial to evaluate an experimental drug. New research at Washington University School of Medicine in St. Louis shows the drug extends survival and reverses some neuromuscular damage in mice and rats and may help people whose disease is caused by mutations in the gene SOD1. (Photo: Mike Worful)

Drug extends survival, reverses some neuromuscular damage in animals

From the WashU School of Medicine News

About 20,000 people in the United States are living with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. The invariably fatal disease kills the nerve cells that control walking, eating and breathing. Few people survive more than three years after diagnosis.

Now, new research led by Washington University School of Medicine in St. Louis indicates an investigational therapy for an inherited form of ALS extends survival and reverses signs of neuromuscular damage in mice and rats. The findings, published July 16 in The Journal of Clinical Investigation, have led to a phase one/two clinical trial to investigate whether the drug could benefit people with ALS whose disease is caused by mutations in a gene called SOD1.

“This drug had an impressive effect in mice and rats with just one or two doses,” said Timothy Miller, MD, PhD, the David Clayson Professor of Neurology at Washington University. “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.”

About 10 percent of ALS cases are inherited. Of those, about a fifth are caused by mutations in SOD1. Such mutations cause the SOD1 protein to be overly active, which suggests that reducing protein levels might help ALS patients with SOD1 mutations.

Patients with ALS have few options for treatment. Only two drugs have been approved by the Food and Drug Administration (FDA) for ALS, and both only modestly slow the course of the disease.

In collaboration with Ionis Pharmaceuticals, Miller and colleagues tested DNA-based compounds that block the body from making SOD1 protein.

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