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May 28, 2026Alzheimer s & Dementia1 citationsOpen Access

Repurposed drug prioritization pipeline for a multi‐arm platform trial in clinical Alzheimer's disease

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SISabahat IqbalCOCristina Bonet OlivaresLRLaura Rizzo

Key Points

  • The aim is to create a systematic method for selecting repurposed drugs for Alzheimer's disease treatment.
  • Developed a drug prioritization pipeline funded by the UK NIHR.
  • Invited drug proposals from the AD community using a standardized template.
  • An international expert panel ranked compounds based on key criteria during multiple meetings.
  • Atomoxetine ranked first, metformin second, and isosorbide mononitrate and levetiracetam tied for third.
  • Compounds were evaluated for biological plausibility, preclinical efficacy, safety, and trial feasibility.

Abstract

INTRODUCTION: There is an urgent need to identify effective, safe, and affordable treatments for Alzheimer's disease (AD). Funded by the UK National Institute for Health and Care Research (NIHR), we developed a systematic drug prioritization pipeline to identify repurposed drug candidates for inclusion in a planned platform trial for clinical AD. METHODS: The wider AD community was invited to propose compounds using a standardized proposal template. Fourteen proposals were presented to an international expert panel, who independently ranked compounds based on biological plausibility, preclinical efficacy, safety, and trial feasibility. Extended drug summaries were compiled for shortlisted compounds, supported by ReLiSyR, a machine learning-supported systematic review tool. RESULTS: Following review in a second panel meeting, independent re-ranking identified the following compounds: atomoxetine (1st), metformin (2nd), isosorbide mononitrate and levetiracetam (joint 3rd). DISCUSSION: This robust drug prioritization pipeline will speed the identification and progression of promising candidates for therapeutic evaluation.

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Cite This Study

Iqbal et al. (2026) studied this question.

synapsesocial.com/papers/6a17dd4e3fad632b0f9da032https://doi.org/10.1002/alz.71495
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