Biogen’s targeted ALS treatment, Qalsody, has shown the potential to reverse disease decline in some patients, according to an Aug. 10 report. Paula Trefiak, a ballet dancer and mother of three from Saskatchewan diagnosed with ALS in 2016, experienced improvement after participating in clinical trials for tofersen—now marketed as Qalsody—developed by Biogen and Ionis Pharmaceuticals.
Trefiak initially received a low dose of the drug during a Phase 1b trial but continued to deteriorate until she was given a higher dose in an open-label extension study. She reported stronger muscles and improved quality of life following the increased dosage. "I started to notice I was having a better sleep," Trefiak said. "I wasn’t constantly waking up in agony for hours on end with cramping." More than ten years after her diagnosis, Trefiak says she can still dance and swim.
Qalsody is currently approved only for patients with ALS linked to mutations in the superoxide dismutase (SOD1) gene—a subset that affects less than 500 people in the United States. The U.S. Food and Drug Administration granted accelerated approval based on biomarker data from the Phase 3 Valor trial in April 2023; Health Canada followed under its Special Access Program two years later.
Angela Genge, chief medical officer at AL-S Pharma, said at least one patient treated with AP-101—a different therapy targeting misfolded SOD1 protein—also saw reversal of decline during clinical trials. Data presented at ENCALS 2026 indicated that early treatment with AP-101 prolonged survival and delayed ventilator use among both familial and sporadic ALS patients showing elevated levels of misfolded SOD1.
Researchers are exploring whether targeting SOD1 could benefit more ALS patients beyond those carrying specific mutations since pathological misfolding occurs even among sporadic cases. Washington University is conducting a Phase 2 study on non-SOD1 ALS patients using Qalsody; primary completion is expected by January 2027.
While Qalsody helps stabilize or improve symptoms for some individuals like Trefiak, it is not considered a cure and requires ongoing intrathecal injections every four weeks. Other therapies such as AP-101 may offer alternative delivery methods if approved.
