CuATSM efficacy is independently replicated in a SOD1 mouse model of ALS while unmetallated ATSM therapy fails to reveal benefits

Fernando G Vieira; Theo Hatzipetros; Kenneth Thompson; Andy J Moreno; Joshua D Kidd; Valerie R Tassinari; Beth Levine; Steven Perrin; Alan Gill

doi:10.1016/j.ibror.2017.03.001

CuATSM efficacy is independently replicated in a SOD1 mouse model of ALS while unmetallated ATSM therapy fails to reveal benefits

IBRO Rep. 2017 Mar 12:2:47-53. doi: 10.1016/j.ibror.2017.03.001. eCollection 2017 Jun.

Authors

Fernando G Vieira¹, Theo Hatzipetros¹, Kenneth Thompson¹, Andy J Moreno¹, Joshua D Kidd¹, Valerie R Tassinari¹, Beth Levine¹, Steven Perrin¹, Alan Gill¹

Affiliation

¹ ALS Therapy Development Institute, USA.

Abstract

A copper chelator known as diacetylbis(N(4)-methylthiosemicarbazonato) copper II (CuATSM), has been reported to be efficacious in multiple transgenic SOD1 models of amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disorder affecting motor neurons. Here we report that we also observed CuATSM efficacy on disease onset and progression in a standardized litter-matched and gender-balanced efficacy study using B6SJL-SOD1G93A/1Gur mice. We also report improved survival trends with CuATSM treatment. In addition, we report a lack of efficacy by unmetallated ATSM in the same model using the same standardized study design. These results add to existing evidence supporting an efficacious role for copper delivery using chaperone molecules in mouse models of ALS.