AI Helps Find a Cause of Alzheimer's Disease and Identify Therapeutic Candidate
a year ago
- #Alzheimer’s Disease
- #Gene Regulation
- #Artificial Intelligence
- A new study identifies the PHGDH gene as a cause of Alzheimer’s disease due to its previously unknown secondary function.
- Researchers at UC San Diego used AI to uncover PHGDH’s role in disrupting gene regulation in brain cells, leading to Alzheimer’s.
- PHGDH’s increased expression correlates with Alzheimer’s progression, and altering its levels affects disease advancement in mice and human brain organoids.
- AI helped visualize PHGDH’s 3D structure, revealing a DNA-binding domain that activates genes, disrupting cellular balance and causing Alzheimer’s.
- The small molecule NCT-503 was identified as a potential therapeutic candidate, inhibiting PHGDH’s regulatory role and alleviating Alzheimer’s symptoms in mice.
- NCT-503 targets an upstream pathway, potentially preventing amyloid plaque formation, unlike current treatments that address plaques after they form.
- The study suggests new classes of small molecules could be developed into future Alzheimer’s therapeutics, possibly administered orally.
- Limitations include the lack of perfect animal models for spontaneous Alzheimer’s, but results are promising for further clinical development.