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Cancer Drugs Reverse Alzheimer’s Pathology in Mice, Advance to Clinical Trials

Researchers are preparing human trials of letrozole and irinotecan after electronic health data linked the combo to lower Alzheimer’s risk, mindful of its side-effect profile

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Letrozole tablets.
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Overview

  • Researchers used large-scale gene expression data to screen over 1,300 FDA-approved drugs for potential to counter Alzheimer’s signature changes.
  • Letrozole and irinotecan emerged as top candidates targeting neuronal and glial pathways disrupted in Alzheimer’s.
  • In Alzheimer’s mouse models, the combination reversed brain degeneration, reduced amyloid and tau buildup, and restored memory.
  • Analysis of millions of de-identified health records showed a lower incidence of Alzheimer’s among patients treated with these cancer drugs.
  • Investigators plan to launch clinical trials to evaluate efficacy in patients while monitoring known side effects such as hot flashes, diarrhea, nausea and vomiting.