The ALS-FTD spectrum has emerged as one of the most active research areas in neurodegeneration. C9orf72 ASO trials are progressing in parallel for both ALS and FTD. Progranulin replacement therapy (AL001) entered Phase III in GRN-FTD patients in 2024. View full research signals →
Pathological hallmark of ~45% of FTD cases. Shared with ALS — the ALS-FTD spectrum is defined by TDP-43 proteinopathy. Emerging plasma assays improving accessibility.
Elevated in FTD CSF and plasma. Correlates with atrophy rate and disease progression. Used in multiple FTD trials as a pharmacodynamic marker.
Astrocytic damage marker. Differentiates FTD from Alzheimer's in plasma — GFAP is lower in FTD than AD, making it a useful differential biomarker.
Unlike Alzheimer's, tau is typically normal or low in FTD CSF. Its absence helps differentiate FTD from tauopathies. Specific tau isoforms elevated in CBS and PSP subtypes.
| NCT ID | Drug / Intervention | Phase | Status | Sponsor |
|---|---|---|---|---|
| NCT04374136 | AL001 (progranulin) — GRN-FTD | Phase III | Recruiting | Alector |
| NCT02929511 | ABBV-CLS-7262 (C9orf72 ASO) | Phase II | Active | AbbVie / Clarus |
| NCT04629365 | Semaglutide (neuroinflammation) | Phase II | Recruiting | Academic consortium |
Phase 1 primary cohort + monitored diseases