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.