Scientists are currently investigating the potential of an oral drug, sprinkled with gold nanoparticles, to treat neurodegenerative diseases such as Parkinson’s and multiple sclerosis. The drug, known as CNM-Au8, has shown promising results in phase II clinical trials, indicating that it can enhance the brain’s metabolism.
Though research on the drug’s safety and efficiency is still ongoing, the initial findings have left researchers optimistic. CNM-Au8 comprises of gold nanoparticles that can pass through the blood-brain barrier, increasing the energy supplied to neurons and preventing their decline.
“We are cautiously optimistic that we will be able to prevent or even reverse some neurological disabilities with this strategy,” says neurologist Peter Sguigna from the University of Texas Southwestern.
Parkinson’s disease and multiple sclerosis (MS) are neurological disorders that manifest symptoms of slow brain metabolism. This is believed to deprive neurons of essential energy, leading to the accumulation of harmful toxins and unstable molecules known as free radicals that can damage cells.
Patients with Parkinson’s and MS exhibit abnormally low NAD+/NADH ratios, which are indicative of the disease state. However, recent clinical trials have shown that when 13 Parkinson’s patients and 11 MS patients took CNM-Au8 every day for 12 weeks or more, their baseline NAD+/NADH ratio increased by an average of 10.4%.
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This improvement is significant, given that some studies suggest that the average person loses about 0.5% of their NAD+/NADH ratio per decade. The researchers claim that this increase in NAD+/NADH ratio is sufficient to reverse the decline in neurodegenerative diseases, not just delay them.
In the trial, participants reported a degree of “improved motor experiences of daily living” while taking the drug. However, it remains unknown how the drug works, although preclinical studies on CNM-Au8 suggest that it penetrates the blood-brain barrier and increases energy production in the form of ATP.
Animal studies have demonstrated that CNM-Au8 can counter some of the symptoms of MS by repairing the sheaths that surround neurons and help them send messages more efficiently.
The UT Southwestern researchers state that CNM-Au8 is the only gold nanocrystal suspension known to have exceptionally high catalytic activity and low toxicity that is being developed as a disease-modifying agent for neurodegenerative diseases.
The potential benefits revealed in phase II clinical trials have led the team to prepare for phase III.
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References:
1. Ren, J., Dewey, R.B., Rynders, A. et al. Evidence of brain target engagement in Parkinson’s disease and multiple sclerosis by the investigational nanomedicine, CNM-Au8, in the REPAIR phase 2 clinical trials. J Nanobiotechnol 21, 478 (2023). https://doi.org/10.1186/s12951-023-02236-z
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