CuATSM (Copper-ATSM) for ALS
Also known as: Cu-ATSM, Copper diacetyl-bis(N4-methylthiosemicarbazone)
A copper-delivering compound that targets SOD1 protein misfolding and mitochondrial copper deficiency in ALS motor neurons.
Mechanism of Action
CuATSM delivers bioavailable copper to SOD1 protein, correcting misfolding caused by copper deficiency in mutant SOD1. It also acts as an antioxidant, scavenging peroxynitrite. In SOD1 mouse models, it dramatically extends survival and improves motor function.
General mechanism: Copper delivery agent. Corrects SOD1 copper deficiency. Peroxynitrite scavenger.
Current Evidence
Remarkable preclinical results in SOD1 mice (extended survival by months). Phase I trial in ALS patients showed safety and tolerability. Phase II/III trials underway. Patient-reported anecdotal benefits have generated significant interest.
Clinical Status: Phase II/III clinical trials ongoing. Breakthrough therapy potential for SOD1-ALS.
Safety Profile
Well-tolerated in Phase I. Copper metabolism monitoring required. Long-term safety data still accumulating.
Key Research Questions
- Does CuATSM benefit non-SOD1 ALS subtypes?
- What is the optimal dosing regimen for CNS copper delivery?
- Can CuATSM be combined with tofersen for enhanced SOD1-ALS treatment?