Wexner Medical Center among first in nation to administer new gene therapy for ALS

Oct. 6, 2023
ALS is a complicated and progressive disorder affecting more than 31,000 people in the United States, according to the Centers for Disease Control and Prevention.

The Ohio State University Wexner Medical Center is one of the first nationwide to administer a targeted gene therapy for patients with a specific form of amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that affects the cells in the brain and spine.

On April 25, the Food and Drug Administration (FDA) approved the use of tofersen, also known as QALSODY, in a small subset of adults with ALS who have a mutation in the superoxide dismutase 1 (SOD1) gene.

Andrew Wurm, 53, of Granville, Ohio, received his first dose during a 15-minute outpatient procedure at Ohio State’s Spine Center. Steve Severyn, MD, associate professor emeritus of anesthesiology at Ohio State, and his team administered the drug via an X-ray-guided lumbar puncture into his spinal fluid.

After the initial loading doses, Wurm, who was diagnosed with ALS in October 2022, will return on a monthly basis to receive the gene therapy.

The Ohio State University Wexner Medical Center release

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