Abstract
Objective The objective of RESCUE-ALS was to evaluate the efficacy and safety of CNM-Au8, a suspension of catalytic gold nanocrystals that enhance cellular energy metabolism, as a disease-modifying treatment for amyotrophic lateral sclerosis (ALS).
Methods Participants were randomized 1:1 (active:placebo). The primary endpoint was the percent change in the summated motor unit index (MUNIX) scores for four selected limb muscles after 36 weeks of treatment. Secondary/exploratory endpoints included respiratory function, ALS disease progression, and quality of life.
Results In total, 49 participants were screened and 45 enrolled (73% limb onset, 27% bulbar). In the CNM-Au8 30mg cohort, there was significant reduction of ALS disease progression (occurrence of death, tracheostomy, or need for non-invasive ventilatory support or gastrostomy tube placement; 37% absolute risk reduction, p=0.02), improved proportion free from > 6-point ALSFRS-R decline (49% vs. 8%; p=0.04, chi-square test), improved quality of life (LS mean difference: 0.9; 95% CI: 0.2 to 1.6; p=0.02). Additionally, there was a trend for improvement in the summated MUNIX score to week 36 (primary endpoint) that was more prominent in limb-onset ALS (LS mean difference: 20.9%, 95% CI: -2.2% to 44.0%, p=0.074), and a trend for improvement of respiratory dysfunction. CNM-Au8 was well-tolerated, and no safety signals were observed.
Conclusions CNM-Au8, in combination with riluzole, was safe and well-tolerated in ALS. CNM-Au8 may provide functional benefit by slowing ALS disease progression.
