Copper-ATSM as a Treatment for ALS: Support from Mutant SOD1 Models and Beyond
Sara Nikseresht, James BW Hilton, Kai Kysenius, Jeffrey R Liddell, Peter J Crouch
Life | MDPI | Published : 2020
The blood-brain barrier permeant, copper-containing compound, CuII(atsm), has successfully progressed from fundamental research outcomes in the laboratory through to phase 2/3 clinical assessment in patients with the highly aggressive and fatal neurodegenerative condition of amyotrophic lateral sclerosis (ALS). The most compelling outcomes to date to indicate potential for disease-modification have come from pre-clinical studies utilising mouse models that involve transgenic expression of mutated superoxide dismutase 1 (SOD1). Mutant SOD1 mice provide a very robust mammalian model of ALS with high validity, but mutations in SOD1 account for only a small percentage of ALS cases in the clinic,..View full abstract
This research was funded by MND Research Australia (Beryl Bayley Fellowship to J.B.H.; Betty Laidlaw MND Research Grant and Jenny Barr Smith MND Research Grant to P.J.C.), FightMND (Translational Research Grant to P.J.C.), and the University of Melbourne.