A new study found that a gene recently recognized as a biomarker for Alzheimer’s disease is a cause due to its previously unknown secondary function. Researchers at the University of California, San Diego, used Artificial Intelligence (AI) to help unravel this mystery of Alzheimer’s disease and discover a potential treatment. In particular, the researchers found that Phosphoglycerate dehydrogenase deficiency (PHGDH) plays a causal role in disrupting gene regulation in the brain.
Alzheimer's disease is the most common cause of dementia, characterized by a progressive decline in memory, thinking, and behavior. It is caused by the buildup of proteins in the brain, leading to the death of brain cells and brain shrinkage over time. Symptoms typically appear in individuals over 65 and may include memory loss, confusion, and difficulty with problem-solving. While there is currently no cure, medications can help manage symptoms and slow the progression of the disease.
About one in nine people aged 65 and older has Alzheimer’s disease, the most common cause of dementia. While some genes, when mutated, can lead to Alzheimer’s, that connection only accounts for a small percentage of all Alzheimer’s patients. Most patients do not have a mutation in a known disease-causing gene; instead, they have “spontaneous” Alzheimer’s, and the causes for that are unclear.
Using AI, the team revealed that PHGDH has a hidden DNA-binding function unrelated to its known enzymatic activity.
This malfunction triggers early Alzheimer’s development, offering a new target for prevention. They also identified a small molecule, NCT-503, that blocks this harmful activity without affecting everyday brain chemistry.
The key facts include:
- Hidden Role of PHGDH: AI revealed PHGDH acts as a DNA-binding disruptor, leading to Alzheimer’s.
- New Therapeutic Candidate: The small molecule NCT-503 blocks the harmful function without impairing normal activity.
- Promising Results: Treated mice improved memory and anxiety, suggesting clinical potential.
The San Diego team thinks this opens the possibility for a potential treatment that obstructs the gene’s moonlighting role. This new upstream target could potentially prevent the build-up of amyloid plaques, offering earlier intervention against Alzheimer’s, and further research will optimize the drug for human clinical testing.
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