A US research team has shown that CNM-Au8, an experimental drug based on gold nanocrystals, is able to reverse brain deficits in patients with Parkinson’s and Multiple Sclerosis by improving energy metabolism in the brain. Here’s how it works and why it is a hope against neurodegenerative diseases.
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Credit: Clene Nanomedicine
And experimental drug based on gold nanoparticles was able to reverse the cerebral deficit in patients affected by Multiple sclerosis e Parkinson’s diseasetwo municipalities patologie neurodegenerative associated with a energy metabolism of the brain compromise. People with Parkinson’s treated with the drug also claimed to have achieved improvements in motor functions related to daily activities. In other words, this treatment – called CNM-Au8 – can have a positive impact on symptoms and on the progression of the pathology. It is still too early to say anything about the actual effectiveness of the experimental drug, however scientists are optimistic that through it they can be prevenute o reversed “some neurological disabilities” triggered by neurodegeneration.
The experimental drug CNM-Au8 was developed and tested by an international research team led by scientists from the University of Texas Southwestern Medical Center, who collaborated closely with colleagues from Instat Clinical Research and the company Clene Nanomedicine , Inc, the latter specializing in the development of gold nanocrystals as therapeutic agents, to be administered orally. But how exactly does the new drug work? As explained by the American university in a press release, the correct functioning of the brain is linked to the continuous contribution ofadenosine triphosphate (ATP) to its cells. In practice, this molecule represents the “fuel” that allows cells to live and carry out their tasks.
With the’aging the brain’s energy metabolism undergoes a natural decline, due to one imbalance which is determined between the two forms of a coenzymenicotinammide adenine dinucleotide (NAD+), the oxidized form, and nicotinamide adenine dinucleotide + hydrogen (NADH), the reduced form. If this metabolic decline is normal with advancing age, in Parkinson’s disease and other pathologies – including Amyotrophic Lateral Sclerosis – the process is much more evident and accelerated. This is precisely where the experimental drug CNM-Au8 comes into play, providing the missing coenzyme to restore energy balance in the brain, through a process of catalysis. To send it to the brain, scientists use gold nanoparticles, which are capable of cross the blood-brain barrier and transport the active ingredient to the target.
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To verify the effectiveness of CNM-Au8, researchers led by Professor Peter Sguigna, professor of Neurology and researcher at the Peter O’ Donnell Jr. Brain Institute of the Texan university, involved in the study 24 patients, 11 with relapsing Multiple Sclerosis and 13 with Parkinson’s disease. The participants, who underwent brain tests to check baseline NAD + /NADH concentrations, took the drug for 3 months, after which they underwent checks again. Well, compared to the beginning of the study, on average the ratio of NAD + /NADH is increased by 10.4 percent, reversing the metabolic deficit linked to neurodegenerative diseases. It may not seem like much, but it is a very significant effect, considering that every decade the average loss is 0.5 percent. The increase was also reflected in an improvement in motor functions in patients with Parkinson’s, as highlighted by them.
“We are cautiously optimistic that we will be able to prevent or even reverse some neurological disabilities with this strategy,” Professor Sguigna said. As also highlighted by preclinical studies, CNM-Au8 also supports the neuroprotection and the remyelination (the loss of myelin sheath is one of the characteristics of multiple sclerosis). One of the most significant aspects lies in the fact that the drug did not trigger serious side effects. I dettagli dello studio “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” sono stati pubblicati sulla rivista scientifica Journal of Nanobiotechnology.