Repurposing niclosamide for the treatment of neurological disorders - PMC
/PMC/2026
Why It Matters
This caught my attention because we're looking at an already-approved drug with known safety data being tested for brain diseases. The multi-target mechanism is interesting — it hits inflammation, mitochondrial function, and protein clearance all at once. But here's the reality check: most of this data comes from cell cultures and animal models. The human evidence for neurological use is essentially nonexistent at this point.
Key Findings
- Niclosamide affects multiple pathways relevant to neurodegeneration: reduces neuroinflammation, improves mitochondrial function, and enhances clearance of toxic protein aggregates in preclinical models
- In animal models of Alzheimer's disease, niclosamide reduced amyloid-beta plaques and improved cognitive function, though these results haven't been replicated in human trials
- The drug crosses the blood-brain barrier poorly in its standard form — researchers are developing modified versions and alternative delivery methods to improve brain penetration
- Current human use is limited to its approved indication (intestinal parasites) at 2g doses; neurological applications would likely require different dosing or formulation
- Safety profile is well-established for short-term use against parasites, but long-term safety data for chronic neurological treatment doesn't exist yet
Read the Paper↗PMC10358648