Game-changing discovery in Parkinson's treatment: Molecular target that stops damage in the brain was found

There is promising news from the medical world against Parkinson's disease, which affects millions of people around the world and progresses with the gradual loss of dopamine-producing neurons in the brain. Researchers at Nanjing Medical University in China found that the carrier protein called "ABCA1", which maintains cholesterol balance in the brain, is one of the main perpetrators in the progression of the disease. The study, the details of which were shared in Nature Neuroscience, proved that nerve cell losses and motor dysfunctions can be prevented if the protein in question is suppressed.

CHOLESTEROL ACCUMULATION IN THE BRAIN CLOSES CELLULAR RECYCLING

Although past research has shown that Parkinson's patients have abnormal cholesterol accumulations in their brain tissues, drug trials to reduce cholesterol production have not been successful at the clinical level. Nanjing Medical University experts determined that the problem was not caused directly by cholesterol production, but by the transport mechanism at the cellular level.

Analyzes of brain tissues and laboratory models of deceased Parkinson's patients showed that the ABCA1 protein in astrocytes, the support cells, secretes excessive amounts of cholesterol. This excess cholesterol entering the neurons accumulates in lysosomes, the cell's waste clearance center, paralyzing the cellular recycling network. As a result of this blockage, "alpha-synuclein" protein clusters, which are the characteristic sign of Parkinson's and poison nerve cells, proliferate out of control.

CELL DEATH WAS STOPPED WITH GENE THERAPY AND MAGNESIUM CITRATE

The research team managed to silence the Abca1 gene through specially designed viruses (AAV) to stop the devastating effect of the disease. In experimental models, it was observed that neuronal losses significantly decreased and movement disorders improved when the gene was disabled.

One of the most striking findings of the study emerged during the screening of already known molecules. Researchers discovered that the compound magnesium citrate effectively suppresses (inhibitors) the ABCA1 protein. Thanks to the magnesium citrate treatment applied, the death of dopamine-producing neurons was prevented.

PAVING THE ROAD FOR CLINICAL TRIALS ON HUMANS

The data obtained offers a brand new strategy in Parkinson's treatment that can go beyond suppressing symptoms and directly change the course of the disease (disease-modifying). Although the protective effect of magnesium citrate and ABCA1 inhibitor drugs has not yet been confirmed in human clinical trials, the concrete successes achieved in animal models lay a strong foundation for human trials in the near future.

Experts evaluate that this new approach, which targets the cholesterol transport line in the brain, may be a breakthrough in the treatment of Parkinson's as well as other neurodegenerative diseases that progress through similar mechanisms.