11/07/2025
Researchers at UC San Diego used artificial intelligence to discover that a gene linked to Alzheimer's disease, which produces an enzyme called PHGDH, might actually be causing the disease rather than just being a sign of it. The AI helped reveal a hidden role of PHGDH: it can switch certain genes on or off in brain cells called astrocytes. This interaction disrupts how the brain controls inflammation and clears waste, potentially triggering Alzheimer’s progression.
Using AI modeling, the team identified a molecule named NCT-503 that can partially block PHGDH’s gene-switching without stopping its important normal functions. When tested in mice with Alzheimer’s-like symptoms, treatment with NCT-503 improved memory and reduced anxiety. The molecule can cross the blood-brain barrier and might be developed into an oral drug, which is a significant step forward.
Although there’s still a long road before this becomes a treatment for people, the study highlights how AI can uncover hidden biological functions and guide the design of new therapies. This approach opens the door to novel classes of drugs that might better control Alzheimer’s by targeting key molecular triggers rather than just symptoms.
While Alzheimer’s remains complex with limited treatments, this discovery offers new hope by combining AI’s power with biology to find innovative solutions that could one day slow or stop the disease.
