Research signals
Research activity in ALS gene-silencing mechanisms has increased substantially since tofersen's 2023 approval. Neuroinflammation and microglial activation appear in 23% of active ALS trials — up from 8% five years ago. View full research signals →
Tracked biomarkers
Neurofilament Light Chain (NfL)Strong evidence
Universal neuronal damage marker. Elevated in CSF and blood. Used as a secondary endpoint in 8 active ALS trials.
IL-6 / TNF-αModerate evidence
Neuroinflammatory cytokines. Elevated in ALS CSF. Cross-disease panel opportunity with MS biomarker work.
TDP-43 (CSF/plasma)Moderate evidence
Aggregation marker specific to ALS and FTD. Emerging assay technology improving detection sensitivity.
SOD1 (CSF)Emerging
Target engagement marker for SOD1-ASO therapies including tofersen. Used to confirm mechanism of action.
Active clinical trials
| NCT ID | Drug | Phase | Status | Sponsor |
|---|---|---|---|---|
| NCT04235492 | Tofersen (SOD1-ASO) | Phase III | Recruiting | Biogen |
| NCT02588677 | Masitinib | Phase III | Recruiting | AB Science |
| NCT04120740 | TDP-43 chaperone | Phase II | Active | Academic consortium |
Related diseases — shared mechanisms
Phase 1 primary cohort + monitored diseases