Artificial intelligence has played a pivotal role in uncovering a potential trigger for Alzheimer’s disease and identifying a promising treatment. Researchers at the University of California, San Diego have found that an enzyme called phosphoglycerate dehydrogenase (PHGDH) may not only serve as a biomarker for Alzheimer’s but could also be a causal factor in the disease’s progression.
Alzheimer’s disease is one of the most prevalent neurodegenerative disorders worldwide, affecting millions of people. Despite decades of research, its precise causes remain elusive. PHGDH was initially noted for its elevated activity in individuals experiencing rapid cognitive decline, but new findings suggest it may be directly involved in the mechanisms driving neurodegeneration.
How AI Helped Identify PHGDH’s Role
PHGDH is primarily involved in amino acid metabolism, but AI-driven analysis has revealed an additional, previously unknown function—it can act as a gene regulator, switching certain genetic pathways on and off. This disruption interferes with the brain’s ability to regulate inflammation and waste clearance, two crucial factors in maintaining neurological health. When PHGDH activity is abnormally high, it may accelerate the accumulation of toxic proteins, leading to the hallmark symptoms of Alzheimer’s.
A New Therapeutic Candidate: NCT-503
To counteract PHGDH’s harmful effects, researchers identified a molecule called NCT-503, which binds to PHGDH and prevents its gene-switching activity. In experimental studies using mouse models, treatment with NCT-503 resulted in notable improvements in memory and anxiety tests, suggesting it could be developed into a potential therapeutic option for Alzheimer’s patients. By targeting the metabolic and regulatory functions of PHGDH, this compound may offer a new pathway for intervention, especially in early-stage cases.
Implications for Alzheimer’s Research
This discovery underscores AI’s growing role in identifying disease mechanisms and accelerating drug development. By analyzing large datasets and predicting molecular interactions, AI has enabled researchers to pinpoint PHGDH as a key player in Alzheimer’s pathology. Future studies will focus on refining the drug candidate, conducting human trials, and further exploring the role of PHGDH in brain function.
Conclusion

AI-driven research has provided valuable insights into Alzheimer’s disease, revealing a previously unknown trigger and a potential treatment. As scientists continue to explore PHGDH’s role, this breakthrough may bring hope for future interventions that could slow or even prevent the disease’s progression. With advancements in AI and molecular biology, researchers are now closer than ever to uncovering strategies that could mitigate one of the most devastating neurological disorders.
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