Unveiled: fresh reason behind Alzheimer's, unearthed by AI
In a significant development for Alzheimer's research, a team of scientists at the University of Bonn, led by Professor Michael J. P. W. Vitek, have discovered a potential new treatment using the drug NCT-503. This breakthrough, yet to be tested in clinical trials, could revolutionise the way we approach Alzheimer's care.
Currently, most Alzheimer's treatments focus on targeting beta-amyloid plaques. However, the new research targets a different pathway: PHGDH (phosphoglycerate dehydrogenase). This gene, it was found, plays a regulatory role in the brain and its overproduction in Alzheimer's patients directly correlates with disease progression.
PHGDH has a unique ability to bind to DNA and control the activation of other genes. Artificial intelligence was instrumental in uncovering this hidden function, something that traditional research methods had missed for decades. The AI analysis revealed that PHGDH has a secret regulatory function, binding to DNA and controlling the activation of other genes.
The PHGDH pathway operates upstream from plaque formation, potentially preventing amyloid plaques from developing. This is a significant finding, as the vast majority of Alzheimer's patients don't have clear genetic mutations like APP, PSEN, or MAPT.
The potential of NCT-503 as a treatment lies in its ability to cross the blood-brain barrier, a crucial factor for reaching brain tissue where the disease process occurs. Laboratory testing confirmed that NCT-503 successfully blocks PHGDH's harmful gene regulation without affecting normal cellular processes.
When tested on mice with Alzheimer's-like conditions, the treated animals showed substantial improvements in memory and reduced anxiety. These improvements were observed in two different mouse models of Alzheimer's disease, further strengthening the potential of NCT-503 as a promising treatment.
The research team is now working to optimise the compound and prepare it for FDA-required safety studies that precede human clinical trials. If successful, NCT-503's potential for oral administration could transform Alzheimer's care by making treatment more accessible and patient-compliant.
While this is an exciting development, it's important to note that NCT-503 has not yet been tested in clinical trials. Further research will be needed to fully understand its potential and safety in humans. Nevertheless, this discovery represents a significant step forward in our understanding of Alzheimer's disease and potential treatment options.
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