Nanocrystalline Gold Treatment Produces Measurable Metabolic Effects in Parkinson Disease and Multiple Sclerosis

Across 2 phase 2 clinical trials, for Parkinson disease (PD; NCT03815916) and multiple sclerosis (MS; NCT03993171), treatment with nanocrystalline gold (CNM-Au8; Clene Nanomedicine, Salt Lake City, UT) resulted in statistically significant changes in brain metabolism. Brain metabolism was measured by the ratio of nonoxidized to oxidized nicotinamide adenine dinucleotide (NAD⁺/NADH). 

Participants with MS or PD treated with nanocrystalline gold for 12 weeks had a mean 10.4% increase in NAD⁺/NADH (P=.037) compared with their own baseline levels. The mean changes from baseline in both the NAD⁺ and NADH fractions of the total NAD pool were concordant with the change in the ratio of NAD⁺/NADH (P=.026). 

“We believe the REPAIR program represents a critical breakthrough for Clene, demonstrating that catalytically active CNM-Au8 improves energy production and utilization in the brains of people with PD and MS,” said Robert Glanzman, MD, FAAN, chief medical officer, Clene. “Using a novel, noninvasive brain imaging approach, the study demonstrated that a key driver of cellular ATP energy production, the ratio of NAD⁺/NADH, was significantly increased in the brains of patients after 3 months of daily CNM-Au8 oral administration. Remarkably, the data also show a significant rebalancing of brain beta-ATP levels in these patients, a metabolic response to treatment that suggests improved ATP energy efficiency. Our next step will be to demonstrate that these brain energetic changes result in clinically meaningful results in patients with PD and MS.”

Treatment with CNM-Au8 was well-tolerated, with all treatment emergent adverse events reported as predominantly mild and unrelated to the study drug. There were no serious adverse events or treatment discontinuations related to adverse events in either study.

NAD⁺ and NADH levels were measured with phosphorous MR spectroscopy before and after 12 or more weeks of daily oral dosing with CNM-Au8. End of treatment results at week 12 were compared to baseline in 24 patients—13 patients with PD and 11 with MS.