Nanocrystalline Gold Treatment of Amyotrophic Lateral Sclerosis Decreases Mortality Risk, Slows Disease Progression

In the phase 2 RESCUE-ALS study (NCT04098406) individuals with amyotrophic lateral sclerosis (ALS) treated with nanocrystalline gold (CNM-Au8; Clene Nanomedicine; Salt Lake City, UT) had improved survival, slowed disease progression, and higher quality of life compared with individuals who received later treatment for a shorter duration.

For those who received nanocrystalline gold vs placebo for 36 weeks followed by at least 84 weeks of open-label treatment, risk of mortality was decreased by 62% (hazard ratio [HR] 0.38; 95% CI: 0.143-1.03) when compared to those who received placebo for the first 36 weeks. 
As previously reported, individuals treated with nanocrystalline gold vs placebo for 36 weeks had slowed disease progression (P=.013). Additionally lower proportion of those treated with nanocrystalline gold had a 6-point decline in the ALS Functional Rating Scale Revised (ALSFRS-R) (P=.035). 

Nanocrystalline gold treatment resulted in improved quality of life assessed by the ALS Specific QOL short-form scale (ALSSQOL-SF), (P=.018) during the 36-week double-blind period that was maintained through 84 or more weeks of treatment in the open-label study.

“As we continue to perform preclinical experiments in collaboration with academic partners and analyze the enriching dataset from RESCUE-ALS, the data consistently highlight CNM-Au8 as an emerging therapeutic option for people with this devastating disease,” said Robert Glanzman, MD, FAAN, chief medical officer, Clene. “We look forward to the next major data readout from the HEALEY ALS platform trial that is expected in the third quarter.”

These data were presented at the European Network to Cure ALS (ENCALS) meeting, June 1-3 in Edinburgh, Scotland. The posters are available on-demand via the ENCALS conference portal and on the Scientific Posters and Presentations section of the Clene website.