Mebendazole for ALS
Also known as: Vermox
Mebendazole's CNS penetration and anti-inflammatory properties make it a candidate for neuroinflammation modulation in ALS.
Mechanism of Action
In ALS, mebendazole's ability to cross the BBB enables direct neuroinflammatory modulation. It inhibits NF-κB-driven microglial activation and may reduce the non-cell-autonomous toxicity that accelerates motor neuron death. Its tubulin-binding properties may also stabilize axonal transport in affected motor neurons.
General mechanism: Benzimidazole anthelmintic. β-tubulin binding, anti-angiogenic (VEGF), NF-κB modulation, BCL-2 family modulation. Good BBB penetration.
Current Evidence
Preclinical rationale based on anti-inflammatory mechanisms. No ALS-specific clinical trials. Safety data from antiparasitic use informs feasibility. Conceptual overlap with masitinib's anti-inflammatory approach.
Clinical Status: No ALS trials. Rationale based on neuroinflammation reduction and BBB penetration.
Safety Profile
Decades of safe antiparasitic use. Hepatotoxicity monitoring at high doses. GI effects. Myelosuppression possible with prolonged use.
Key Research Questions
- Can low-dose mebendazole safely reduce neuroinflammation in ALS patients?
- Does mebendazole's tubulin stabilization benefit ALS axonal transport?