Exploring the disease-modifying effects of EH301 in mouse models of ALS

Elysium Health have developed EH301, an experimental therapy based on research in NAD+ repletion and sirtuin activation. EH301 was efficacious in an ALS pilot trial; the results of which have initiated a subsequent placebo-controlled trial of EH301 in up to 150 ALS patients. In order to better understand the effects of EH301 and optimize treatment, we now need to investigate the precise mechanisms of action to inform the clinical development of this novel therapeutic approach. In this application, we propose to explore the disease- modifying effects of EH301 in two preclinical animal models of familial ALS and in primary fibroblasts from sporadic ALS patients, focusing on the mechanistic targets of the drug, to support mitochondrial function.