Tofersen treatment did not lower blood SOD1 activity in a study of people with SOD1-related ALS
Original source
Blood SOD1 Activity in ALS Patients Receiving Tofersen Treatment. (opens in a new tab)Compass summarised this from the study's abstract.
Study details
- Studied in
- Human
Population inferred from the title and abstract by Compass.
Related topics
Blood SOD1 activity was lower in people with SOD1 mutations, including some without symptoms, than in control participants and other ALS groups. In 18 people receiving tofersen, SOD1 protein and neurofilament levels fell, but blood SOD1 activity remained stable. The findings suggest tofersen’s treatment effect does not depend on lowering SOD1 activity.
Why this matters
The results are relevant to people with SOD1-related ALS and to clinicians monitoring tofersen treatment. They do not show that tofersen improves outcomes for an individual, and they do not change treatment guidance. The study measured blood activity; whether tofersen affects SOD1 activity in cerebrospinal fluid still needs to be established.
Limitations and context
The treatment analysis included only 18 people. The study assessed blood samples, not cerebrospinal fluid, and the source says further studies are needed to assess cerebrospinal-fluid SOD1 activity. The reported findings come from one journal article and do not by themselves establish clinical benefit or harm.