CuATSM improves motor function and extends survival but is not tolerated at a high dose in SOD1^G93A mice with a C57BL/6 background.

ABSTRACT: The synthetic copper-containing compound, CuATSM, has emerged as one of the most promising drug candidates developed for the treatment of amyotrophic lateral sclerosis (ALS). Multiple studies have reported CuATSM treatment provides therapeutic efficacy in various mouse models of ALS without any observable adverse effects. Moreover, recent results from an open label clinical study suggested that daily oral dosing with CuATSM slows disease progression in patients with both sporadic and familial ALS, providing encouraging support for CuATSM in the treatment of ALS. Here, we assessed CuATSM in high copy SOD1^G93A mice on the congenic C57BL/6 background, treating at 100 mg/kg/day by gavage, starting at 70 days of age. This dose in this specific model has not been assessed previously.

SUBMITTER: Lum JS 

PROVIDER: S-EPMC8481268 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature