Nanocrystalline Gold for Treatment of Amyotrophic Lateral Sclerosis Suggests Clinical Benefits

In the phase 2 RESCUE-ALS clinical trial (NCT04098406), individuals early amyotrophic lateral sclerosis (ALS) who were treated with nanocrystalline gold for 12 weeks had improvement in their Motor Unit Number Index (MUNIX) compared with placebo. In a prespecified subset of participants with limb-onset ALS, MUNIX number and forced vital capacity (FVC) improved after 12 weeks and 36 weeks of treatment. However, no percent change in the sum total of MUNIX from baseline to week 36, which was the primary outcome measure, was observed.

Clinically relevant exploratory endpoints through week 36 demonstrated significant benefits of nanocrystalline gold vs placebo, including slower disease progression, a smaller proportion of participants with a 6-point decline on the ALS Functional Rating Scale Revised (ALSFRS-R), and improved quality-of-life, as measured by the ALS Specific Quality of Life (ALSSQOL-SF).  

“These data are very encouraging to us in the ALS research and treatment community, as they demonstrate clinical benefits with CNM-Au8 treatment in outcomes that matter to patients and provide evidence for improved long-term survival,” said Matthew Kiernan, AM MBBS(Hons), PhD, DSc, FRACP, FAHMS, Bushell Chair of Neurology, University of Sydney. “RESCUE-ALS was a proof-of-concept trial intended to establish that treatment of neuronal energetic failure can provide disease-modifying effects in ALS. I am pleased to see the potential effectiveness of CNM-Au8 demonstrated in this trial, and it is important to confirm these results in a larger clinical trial.”

The HEALEY-ALS platform trial (NCT04297683) evaluating multiple potential agents to treat ALS, including nanocrystalline gold, is now fully enrolled. 

Nanocrystalline gold was well-tolerated with no reported serious adverse events related to treatment. Treatment-emergent adverse events were predominantly mild-to-moderate in severity and most frequently were aspiration pneumonia and transient gastrointestinal distress.  

RESCUE-ALS was a 36-week, randomized, placebo-controlled trial that enrolled 45 patients with early ALS and randomized 1:1 to treatment with nanocrystalline gold at 30mg daily or matching placebo on top of standard of care.