Nanocatalytic Gold Therapy Well Tolerated in Clinical Trial for Multiple Sclerosis 

Preliminary data from the randomized placebo-controlled phase 2 VISIONARY-MS trial (NCT03536559) of nanocatalytic gold therapy (CNM-Au8; Clene Nanomedicine, Salt Lake City, UT) suggest the drug is well tolerated. Preliminary blinded data also suggest that treatment with nanocrystalline gold improves disability measures for people with multiple sclerosis (MS).

For the first 34 enrolled participants, median improvements were seen in low contrast letter acuity (LCLA) and the Multiple Sclerosis Functional Composite (MSFC) subscales (Symbol Digit Modalities Test, 9-Hole Peg Test, and Timed 25-foot Walk (gait). Physiologic measures are also being studied, including retinal cell ganglion layer thickness on optical coherence tomography (OCT) and visual evoked potential latency.

The trial is still enrolling participants and opened 8 study centers in North America. Participants may continue taking disease-modifying treatments while participating in the study. Key enrollment critera are: 

• Age 18 to 55 years (inclusive)
• A clinical diagnosis of relapsing MS made within the last 15 years using the McDonald criteria
• Best-corrected High Contrast Visual Acuity deficit of 20/200 or less
• Best corrected LCLA impairment of 20/40 using the 6/12 metric or worse in the affected eye AND 20/32 (6/9.5 metric) or worse in the fellow eye
• Mean RNFL thickness ≥ 70 μm in both eyes
• No relapses within the prior 3 months
• No acute optic neuritis within prior 6 months

Robert Glanzman, MD, Clene's chief medical officer said,"These data add to the growing body of clinical evidence demonstrating that CNM-Au8, a suspension of catalytic, clean-surfaced, faceted gold nanocrystals, has the unique ability to improve remyelination and provide axonal neuroprotection. The consistent median improvements observed across the MSFC functional domains in the population of participants in VISIONARY-MS are exciting. We are pleased to be continuing enrollment in the VISIONARY-MS clinical trial and encourage clinicians to consider referring patients with visual impairment to the trial."

No serious adverse events related to treatment have been seen to date and available data suggest that nanocrystalline gold is well-tolerated, with most adverse events being mild and consisting of headache, upper respiratory infection, and sore throat. 

As reported previously in Practical Neurology, it is thought that nanocrystalline gold improves efficiency of bioenergetic reactions and reduces free radicals known to cause oxidative stress. By providing increased energy efficiency and reducing free radicals, nanocrystalline gold may promote remyelination and axonal regeneration.

Nanocrystalline gold is being tested for potential treatment of amyotrophic lateral sclerosis and Parkinson disease in addition to MS. 
These data were presented at the Joint NAIMS-IMSVISUAL Symposium in conjunction with the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum 2020, February 27-29 in West Palm Beach, Florida.