Gold nanocrystals may improve brain metabolic profile

Interim results from two phase 2 studies provide evidence that orally delivered CNM-Au8 (gold nanocrystals) have catalytic effects on key bioenergetic metabolites in the brain of patients with MS or Parkinson’s disease (PD) [1]. The observed metabolic homeostasis may be neuroprotective.

CNM-Au8 is a suspension of clean-surfaced, faceted, gold nanocrystals that promotes remyelination and neuroprotection by catalysing nicotinamide adenine dinucleotide (NAD) and adenosine triphosphate (ATP) production and by reducing oxidative stress. Treatment effects are evaluated using high-resolution ³¹P-magnetic resonance spectroscopy (³¹P-MRS). With this quantitative technique, bioenergetic metabolites can be monitored, as well as phospholipid and myelin precursors.

Preliminary target engagement data from 2 ongoing open-label imaging trials were presented: REPAIR-MS and REPAIR-PD. Treatment consisted of CNM-Au8 30 mg/day. 7T ³¹P-MRS was conducted at baseline and after 12-16 weeks of treatment. Results of 4 completers with MS and 6 with PD were available.

For key bioenergetic markers, percentage change from baseline at the end-of-study visit highly correlated with baseline levels. Whole-brain baseline NAD levels below the mean significantly increased at end-of-study visit. However, baseline NAD levels higher than the mean normalised to mean baseline levels. These dynamics were not only seen for total NAD levels (r²=0.6384; P=0.0056), but also for β-ATP (r²=0.8723; P<0.0001), and several other ³¹P-metabolites.

According to the authors, these observations indicate that CNM-Au8 has a homeostatic effect on brain bioenergetics. Full analyses will be conducted once the 2 trials are completed. Another ongoing study, called VISIONARY-MS, will further explore the possible neuroprotective effects of the brain metabolic homeostasis observed with CNM-Au8 treatment.

1. Ren J, et al. Effects of nanocatalysis on CNS bioenergetic markers in patients treated with CNM-Au8: Interim results from two Phase 2 ³¹Phosphorous imaging studies. MSVirtual 2020, P0206.