DALLAS — In an exciting medical development, phase two clinical trials have shown promising results for patients with multiple sclerosis (MS) and Parkinson’s disease. Researchers from UT Southwestern Medical Center have discovered that a daily intake of gold nanocrystals can significantly reverse disease-related energy deficits in the brain, leading to functional improvements among patients. This innovative approach could potentially offer new hope to individuals suffering from these debilitating neurodegenerative diseases.
At the heart of healthy brain function lies the molecule adenosine triphosphate (ATP), which serves as the primary energy carrier in all living cells. As we age, our brain’s ability to metabolize energy declines, marked by a decrease in the ratio of nicotinamide adenine dinucleotide (NAD+) and its reduced form, NADH. This decline is far more rapid and severe in neurodegenerative conditions like MS, Parkinson’s, and amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig’s disease.
“We are cautiously optimistic that we will be able to prevent or even reverse some neurological disabilities with this strategy,” says Peter Sguigna, M.D., who leads the active MS trial and is an Assistant Professor of Neurology and an Investigator in the Peter O’Donnell Jr. Brain Institute at UT Southwestern, in a media release.
The trials focused on an experimental treatment named CNM-Au8, developed by Clene Nanomedicine. This treatment consists of gold nanocrystals that act as catalysts to improve the brain cells’ energy balance by enhancing the NAD+/NADH ratio. This effect has been demonstrated in cellular and animal models in previous studies, suggesting a potential breakthrough in treating neurodegenerative diseases.
💡What Is Parkinson’s Disease?
- Parkinson’s is a chronic neurological condition where nerve cells in the brain deteriorate.
- This leads to a decrease in dopamine, a vital chemical for movement control.
- The most well-known symptoms include tremors, slowness of movement (bradykinesia), stiffness (rigidity), and balance issues.
- Parkinson’s worsens over time, but the pace and symptoms vary greatly from patient to patient.
- There is currently no cure for the disease.
To verify the effectiveness of CNM-Au8 in humans, researchers conducted two phase two clinical trials, REPAIR-MS and REPAIR-PD, involving 11 participants with relapsing MS and 13 with Parkinson’s disease. The study used brain magnetic resonance (MR) spectroscopy scans to measure the patients’ baseline NAD+/NADH ratios and levels of other molecules associated with cell energy metabolism. After a 12-week daily dose of CNM-Au8, a second MR spectroscopy revealed an average increase in NAD+/NADH ratios of 10.4 percent among the 24 patients, indicating the treatment’s direct impact on the brain.
Moreover, other energy-related molecules, including ATP, normalized, pointing to another potentially beneficial effect of CNM-Au8. Notably, patients with Parkinson’s reported improved motor experiences in daily living, suggesting that the treatment could alleviate functional symptoms of the disease. Importantly, none of the patients experienced severe adverse side-effects linked to the gold nanocrystal treatment.
In the case of Parkinson’s disease specifically, the breakthrough offers new hope to the roughly 500,000 people living with Parkinson’s in the United States alone. Following Alzheimer’s disease, Parkinson’s is the second-most common neurodegenerative disorder in the U.S. However, the National Institutes of Health estimates that up to one million Americans may be living with Parkinson’s — with half of them not knowing it yet.
Although most patients are diagnosed with Parkinson’s disease after the age of 50, the neurodegenerative condition can strike even earlier. The most famous example of this is actor Michael J. Fox, who was diagnosed with Parkinson’s at age 29. Despite the incurable nature of the condition, Fox has not let it defeat him, as he recently took the stage at the 2024 BAFTAs, now at age 62.
While these findings are encouraging, further research is necessary, as Dr. Sguigna notes, to determine whether these results can be replicated in progressive forms of MS. The study’s significance is further underscored by contributions from other UT Southwestern researchers, including Jimin Ren, Ph.D., who led the MR spectroscopy portion, and Benjamin Greenberg, M.D.
This study received its funding from Clene Nanomedicine. When the trials began, Dr. Greenberg was solely affiliated with UTSW. He was employed by Clene Nanomedicine as a consultant after the conclusion of part one of REPAIR-MS.
The findings are published in the Journal of Nanobiotechnology.