Nanocrystalline Gold Provides Functional Improvements for Relapsing Multiple Sclerosis

In the VISIONARY-MS clinical trial (NCT03536559), participants with relapsing multiple sclerosis (MS) and optic neuritis (ON) treated with nanocrystalline gold (CNM-Au8; Clene Inc, Salt Lake City, UT) vs placebo had improvements in vision, function, and biomarkers of disease progression. 

Enrollment in this phase 2 proof-of-concept 48-week trial was stopped prematurely, owing to the COVID-19 pandemic, when 73 participants had enrolled. At that time, the benchmark for statistical significance of effect was adjusted to P=.10. Participants were treated with 15 or 30 mg/day nanocrystalline gold or placebo. 

Using a modified intention-to-treat population, those treated with nanocrystalline gold vs placebo had a 3.13-point improvement on the low-contrast letter acuity (LCLA) test in their clinically affected eye (least squares mean difference (95% CI: -0.08 to 6.33, P= 0.056). On the Multiple Sclerosis Functional Composite (MSFC), there was a 0.28-point difference (95% CI: 0.04, 0.52, P= .0207) favoring nanocrystalline gold treatment. 

Improvements in electrophysiologic and imaging biomarkers of ON, including multifocal visual evoked potential (mfVEP) amplitude and latency, retinal thickness on optical coherence tomography (OCT), optic nerve magnetization transfer ratio (MTR) and diffusion tensor imaging (DTI) were also observed.

“These data are very encouraging to us in the MS research and treatment community as we work to address functional improvement in patients,” said Benjamin Greenberg, MD, MHS, FANA, FAAN, CRND professor of Neurology and a clinical advisor for the trial. “The MS community has been successful at limiting relapses, but we need therapies to address progression independent of relapse activity (PIRA). This study was designed as a proof-of-concept evaluation to establish that treatment of neuronal and glial energetic failure can support remyelination and neuroprotection in people living with MS. I am pleased to see the potential effectiveness of CNM-Au8 demonstrated in this trial.”