Researchers may have hit "gold" when it comes to the treatment of multiple sclerosis.
An experimental medication called CNM-Au8 — a drinkable liquid with gold nanocrystals — has shown promising results in clinical trials in terms of improvements in MS symptoms.
The "catalytically active" liquid, developed by Clene Nanomedicine in South Carolina, can cross the blood-brain barrier to help improve cellular energy and restore neurological function, according to researchers.
Doctors at the University of Sydney presented the Phase 2 clinical trial findings at the American Academy of Neurology’s annual meeting earlier in April 2024.
The clinical trials included 78 patients who had relapsing multiple sclerosis.
The gold liquid suspension was found to have a "profound clinical benefit," with the patients experiencing physical improvements not achieved in prior trials.
Multiple sclerosis is an autoimmune disease of the central nervous system, brain, spinal cord and optic nerve.
In people with MS, white cells called lymphocytes infiltrate the central nervous system and trigger inflammation.
That often causes them to feel off balance or lose their vision, according to Dr. Robert C. Sergott, chief of the neuro-ophthalmology service at Wills Eye Hospital and professor of ophthalmology, neurology and neurosurgery at Sidney Kimmel Medical College in Philadelphia.
The MS medications that are currently available work by addressing inflammation — but there is a subset of patients that continue to experience symptoms even without inflammation, he said. This is a condition called progression independent of relapse activity (PIRA).
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"This is a non-inflammatory component of the disease, where patients would worsen in vision and cognitive abilities," Sergott, who was involved in the clinical trial for CNM-Au8, told Fox News Digital.
Researchers suspected that something to do with mitochondria — parts of cells that are responsible for supplying those cells with energy — was causing these patients’ symptoms.
"The theory was that if we could give the mitochondria an extra boost, the neurons, axons and other cells in the central nervous system may work better," said Sergott.
"In other words, maybe these cells aren't dead, but they're hibernating."
Gold nanoparticles were originally used to treat rheumatoid arthritis many years ago, according to Sergott.
"Clene shaved the gold particles and [got] them to a very highly purified nanoparticle state — into very small particles, so they can get through the blood-brain barrier to the cells that need it to work better."
Among the 78 participants in the randomized clinical trial performed in Australia, two-thirds of them received the gold treatment and the other third received a placebo over a three-year period.
"We had hoped to enroll more patients, but COVID intervened," Sergott said.
Neither the patients nor the neurologist overseeing the trial knew who was receiving the actual medicine.
"Patients saw a clinical improvement in the function of their vision and their cognitive ability," Sergott reported.
Doctors also noted an improvement in the electrophysiology, he said — "the patients’ MRIs looked better, which is a special measure."
The patients who received the medicine had no decline in retinal thickness, but those on the placebo did see a decline, he said.
"This was a very significant result, and gives us a lot of hope that we may be able to help patients who have deficits from MS and have had progression independent of relapse activity — or maybe they had an attack and didn't get complete recovery from it," Sergott said.
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None of the trial participants reported any adverse side effects, he said.
"The safety profile is excellent," Sergott told Fox News Digital. "I can't say this doesn't have any side effects — but no patients discontinued the trial because of a side effect that was significant."
With the successful Phase 2 trials complete, researchers are now looking ahead to Phase 3 trials.
"We're very encouraged and we're ready to take the next step," said Sergott. "It's really going to help a lot of people."
Patients taking CNM-Au8 would still need to continue with the standard regimen of anti-inflammatory medications, the doctor noted.
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Looking ahead, there is the potential for the "liquid gold" medication to help treat symptoms of other neurological disorders, including amyotrophic lateral sclerosis (ALS), Alzheimer’s and Parkinson’s, according to Sergott — but the focus is on MS for now.
"We don't want to get too far ahead of ourselves, but there is plenty of information to support trying this in other diseases."
Damian Kunko of Arlington, Virginia, was one of the MS patients who participated in the trial. He had previously taken disease-modifying therapies that included Zeposia and Tecfidera.
"These drugs reduced the risk of relapse, but had no effect on progressive symptom worsening," Kunko told Fox News Digital.
Kunko’s primary symptoms included heat intolerance, foot drop/limping, numbness, balance issues, double vision and minor decline of cognitive function and energy.
After four months of taking CNM-Au8, Kunko found that his walking speed was 14% faster.
"I was also able to do short 5-10-yard jogs without falling, which was impossible prior to taking [the drug]," he said.
"I also experienced improved visual acuity, less severe double vision, zero cognitive fog issues and increased energy levels."
Later in the trial, Kunko was able to withstand high heat and humidity for several hours and did not experience any double vision, foot drop, excessive numbness or fatigue issues.
"It was nice to be able to go up and down stairs without having to hold the railing," he said.
The patient said he did not experience any negative side effects or adverse reactions.
"The best ‘side effect’ was knowing that this drug will fix my MS issues," Kunko said.
"I am very disappointed that this therapy is not yet commercially available for those who need to restore function and alleviate MS symptoms," he added.
Dr. Marc Siegel, clinical professor of medicine at NYU Langone Medical Center and a Fox News medical contributor, was not involved in the drug’s development but commented on the potential of gold therapies.
"Gold-containing analogies have long been treatments for rheumatoid arthritis — what is known as disease-modifying anti-rheumatic drugs," he told Fox News Digital.
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"And now gold-containing agents are being studied in MS and ALS."
While the latest study is small, Siegel said, it "looks promising in terms of modifying disease and improving thinking and vision, which can be affected in the early stages of MS."
He added, "More research with larger numbers needs to be done to be conclusive."
Dr. James Kuo, vice president of R&D at Silo Pharma in New Jersey, was also not involved in the drug’s development but said the trial results are "encouraging."
"These new long-term results from the Phase 2 clinical trial support the therapeutic hypothesis that CNM-Au8 is remyelinating nerve cells in MS patients," Kuo told Fox News Digital.
"What is further encouraging is that both the primary and secondary endpoints showed continued improvement," he added. "There was physiological enhancement in the visual neural pathway, a biomarker of nerve health. Further MRI measurements support remyelination occurring."
"If further clinical data supports these initial findings, MS patients could well have a new, well-tolerated oral therapy that is not based upon immune system modulation."
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