New results show promise in treatment of SOD1-ALS

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New results from the Phase 3 clinical trial of tofersen, a drug in development by Biogen, have shown promise in treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).

According to the 12-month data, earlier initiation of treatment with tofersen slowed declines in clinical and respiratory function, muscle strength, and quality of life compared to delayed treatment with the drug. Building on earlier trials, this Phase 3 VALOR (placebo-controlled) trial and open-label extension showed that reductions in key biomarkers were sustained over time, demonstrating the drug’s potential to slow the progression of SOD1-ALS.

The results were presented today (June 3) at the European Network to Cure ALS (ENCALS) meeting in Edinburgh, Scotland.

“We are excited to see these results and proud to have supported this clinical trial through the Les Turner ALS Center at Northwestern Medicine,” said Andrea Pauls Backman, CEO of the Les Turner ALS Foundation. “We are grateful to the people living with ALS who participated, both in Illinois and across the world, and to Biogen for pursuing additional data in this important trial. Like them, we are committed to providing hope and improving quality of life for people living with ALS.”

Co-discovered by Teepu Siddique, MD, DSc at Northwestern Medicine, SOD1 was the first gene mutation discovered in ALS. SOD1 is a gene in the human body that instructs cells on how to break down toxic molecules known as superoxide radicals. Breaking down these molecules is an important biological process that protects the cells from damage.

However, mutations in the SOD1 gene have been found to cause a form of ALS by altering an enzyme that is involved in that process, which triggers the death of nerve cells that control muscle movement. Representing about two percent of all cases of ALS globally, SOD1-ALS is associated with a shorter life expectancy compared to other forms of ALS caused by genetic mutations.

Senda Ajroud-Driss, MD, is Associate Professor of Neurology at Northwestern Medicine and Director of the Lois Insolia ALS Clinic at the Les Turner ALS Center at Northwestern Medicine, which was one of the sites for the clinical trials. As a principal investigator of the study, Dr. Driss led the administration of the investigational drug at the Les Turner ALS Center, observation of trial participants, and reporting of results, ensuring rigorous adherence to federal regulations and study protocol requirements.

Offering access to enrollment in multiple clinical trials and with dedicated clinical trial coordinators, the Lois Insolia ALS Clinic at the Les Turner ALS Center is Chicagoland’s first and largest multidisciplinary ALS clinic. It provides access to the highest number of neurologists and dedicated pulmonologists in the Midwest as well as coordinated support and expertise at no cost from the Les Turner ALS Foundation.

The ATLAS Phase 3 trial of tofersen, whose sites include Northwestern University, is recruiting participants. Visit to learn more about ALS clinical trials and support services from the Les Turner ALS Foundation.

Pictured: Dr. Senda Ajroud-Driss providing patient care at the Les Turner ALS Center at Northwestern Medicine