Breakthrough Study: Cancer Drugs Show Promise in Reversing Alzheimer’s Effects

UCSF Researchers Use Big Data to Identify Existing Medications That May Combat Dementia

A major breakthrough out of UC San Francisco (UCSF) and the Gladstone Institutes has revealed a bold new possibility in the fight against Alzheimer’s: repurposing cancer drugs to restore memory and reduce brain degeneration.

Published in Cell (July 2025), this innovative study is shaking up how researchers approach Alzheimer’s treatment. Rather than developing a brand-new drug from scratch, scientists used big data and artificial intelligence to identify two existing FDA-approved cancer drugs—letrozole and irinotecan—that appear to reverse Alzheimer’s effects in lab models.

The Discovery: Flipping Alzheimer’s Gene Activity with Existing Medications

The researchers began by mapping how Alzheimer’s disease alters gene expression in the brain. They then used AI-driven analysis to comb through thousands of approved drugs, looking for compounds that would “flip” that gene signature back to a healthy state.

Two unlikely candidates stood out: letrozole and irinotecan, both originally designed to fight cancer. When used together in Alzheimer’s mouse models, the results were remarkable:

• 🧠 Reduced brain degeneration
  
  
• 🧠 Restored cognitive function and memory
  
  
• 🧠 Marked protection against tau protein buildup
  
  

While each drug individually had limited impact, the combination produced powerful synergistic effects—demonstrating the brain’s remarkable potential for repair, even in late-stage disease models.

Why This Research Could Accelerate Alzheimer’s Treatment

One of the most promising aspects of this discovery is speed. Because letrozole and irinotecan are already FDA-approved, they’ve passed rigorous safety trials. This means:

• 💊 Faster transition to clinical trials in humans
  
  
• 🧬 Reduced cost and risk of development
  
  
• 🧠 Increased potential for near-term treatment options
  
  

Millions of people and their families are waiting for real progress in Alzheimer’s care. Repurposing proven drugs could provide a shorter path to relief, compared to developing entirely new compounds that can take over a decade to approve.

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