Scientists Discover Hidden Cause of Alzheimer’s Hiding in Plain Sight – SciTechDaily

A new study by UC San Diego researchers has shown that the gene PHGDH, once considered only a biomarker for Alzheimer’s, actually plays a direct causal role by disrupting gene regulation in the brain through a newly discovered, non-enzymatic function. Using AI, they identified a drug candidate, NCT-503, that targets this “moonlighting” function and significantly slowed Alzheimer’s progression in mouse models. Credit: SciTechDaily.com

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Scientists Discover Hidden Cause of Alzheimer’s Hiding in Plain Sight
By University of California – San DiegoMay 10, 2025, 9 Comments, 7 Mins Read

A new study by UC San Diego researchers has shown that the gene PHGDH, once considered only a biomarker for Alzheimer’s, actually plays a direct causal role by disrupting gene regulation in the brain through a newly discovered, non-enzymatic function. Using AI, they identified a drug candidate, NCT-503, that targets this “moonlighting” function and significantly slowed Alzheimer’s progression in mouse models. Credit: SciTechDaily.com
Researchers found the PHGDH gene directly causes Alzheimer’s and discovered a drug-like molecule, NCT-503, that may help treat the disease early by targeting the gene’s hidden function.

A recent study has revealed that a gene previously identified as a biomarker for Alzheimer’s disease is not just a marker, it is a direct cause of the disease. Researchers at the University of California, San Diego discovered that the gene plays a previously unrecognized secondary role that actively drives the development of Alzheimer’s. Using artificial intelligence, the team was able to uncover this hidden function and identify a potential therapeutic strategy to block the gene’s harmful activity.

The findings were published on April 23 in the journal Cell.

Alzheimer’s disease affects approximately one in nine people aged 65 and older, making it the most common form of dementia. Although certain genetic mutations are known to cause Alzheimer’s, these cases represent only a small fraction of the total. Most individuals with Alzheimer’s do not carry mutations in any of the established disease-causing genes. These sporadic or “spontaneous” cases have long puzzled scientists, as their underlying causes remain largely unknown.

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