CADASIL Global Research Progress
Last updated: June 2026
β οΈ This report is for educational purposes only and does not constitute medical advice. Always consult your physician.
π Therapeutic Pipeline
| Candidate | Team | Stage | Mechanism | Notes |
|---|---|---|---|---|
| HM101 (Adrenomedullin) | NCVC / Ihara | Phase II Complete | Vascular protection | Japan-based; good safety profile |
| Disulfiram / Auranofin | Wang (Michigan) | In Vitro | NOTCH3 conformational repair | FDA-approved drugs; repurposing |
| PDE5 Inhibitors | Zhao X | In Vitro | Restore NO signaling | Already marketed; fastest path |
| ALT001 | Darwin Origin (Beijing) | Pre-trial (n=6) | Undisclosed | First China-based CADASIL therapy |
| Piezo1 Channel Modulators | Harraz (UVM) | Preclinical | Endothelial Piezo1 correction | 3-5 years to clinic |
| ASO Exon Skipping | Karolinska | Preclinical | Gene silencing | 5-8 years to clinic |
| Vitamin E (T3CAD) | Chabriat | RCT Negative | Antioxidant | Confirmed ineffective |
Data as of June 2026. For reference only.
π¬ Key Research Areas
Drug Repurposing
The fastest path to treatment β using already-approved drugs that show efficacy against CADASIL mechanisms. PDE5 inhibitors, cilostazol + ISMN (LACI-2 trial), and disulfiram are leading candidates.
Gene Therapy
The ultimate cure direction β ASO exon skipping (Karolinska) and CRISPR gene editing. Main challenge: delivery across the blood-brain barrier to cerebral vasculature. 5-10 years away.
Biomarkers
Retinal OCT imaging shows promise as a non-invasive CADASIL biomarker. Blood-based markers (NfL, GFAP) being validated. Critical for clinical trial endpoints.
Patient-Reported Outcomes
2026 international survey (226 patients, 25 countries): fatigue most common (86.3%) and most bothersome symptom. Stress as top trigger. Informing future trial design.
π¬ Educational Videos
cureCADASIL Webinar: NOTCH3 Small Molecule Inhibitors
Dr. Michael Wang, May 28, 2026 β Latest findings on drugs that repair NOTCH3 protein conformation.
Watch on cureCADASIL.org βNIH CADASIL Disease Discovery Study
Dr. Elisa Ferrante, June 2, 2026 β Progress report on the NIH natural history study.
Watch on cureCADASIL.org βVASCERN Patient Education Series
European Reference Network for Rare Vascular Diseases β patient-friendly educational content.
Browse VASCERN resources βUnderstanding CADASIL (cureCADASIL)
Introductory educational video covering basics of CADASIL for newly diagnosed patients and families.
Search on YouTube βπ How to Participate in Research
Find Clinical Trials
- βClinicalTrials.gov (Global)
- βjRCT (Japan)
- βEU Clinical Trials Register
Patient Registries
- βNIH Natural History Study (US)
- βCERVCO Registry (France)
- βContact cureCADASIL for guidance