Lessons in patience gained from the tofersen clinical trials

Pamela Shaw, DBE, MBBS, MD, FRCP, FMedSci, FAAN, FANA, FAAAS, University of Sheffield, Sheffield, UK, evaluates lessons learned from the clinical investigation of tofersen, a drug designed to treat patients with amyotrophic lateral sclerosis (ALS) associated with a genetic mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). She explains that whilst biomarkers in patients’ blood and cerebrospinal fluid demonstrated a reduction in SOD1 and neurofilament protein within six months of the 28-week Phase 3 VALOR study of tofersen (NCT02623699), improvements in motor function were not observed until the trial was extended to 52 weeks. This interview took place at the 18th Annual Congress on Controversies in Neurology (CONy 2024) in London, UK.
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