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152 Repurposed drug prioritisation pipeline for an alzheimer’s disease multi-arm trial

jnnp · 2025-11-26 · canonical JSON source

11 visible annotations · policy: published · automated confidence ≥ 75.00%

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Despite recent developments in amyloid-clearing therapies for Alzheimer’s Disease (AD), there remains a need for more effective treatments. Funded by the UK National Institute of Health and Care Research, we aimed to establish a robust, systematic, and unbiased drug prioritisation pipeline to identify repurposed drugs with the greatest potential for inclusion in a platform trial for clinical AD.We invited the wider AD community to propose compounds by summarising their rationale and evidence in a prespecified drug proposal form. Fourteen proposals were presented to an international expert panel comprising AD clinicians, scientists, industry professionals, and both charity and patient/public representatives. Compounds were ranked on the basis of efficacy, biological plausibility, safety, and feasibility in a platform trial.We compiled an extended drug CV for each shortlisted compound. This incorporated clinically relevant information and was supplemented by systematic clinical literature review using Repurposing Living Systematic Review (ReLiSyR), an automated tool that searches databases and screens studies using machine learning to identify relevant publications.Drug CVs were reviewed in a second panel meeting and compound ranking repeated, as follows: Atomoxetine (1st), Metformin (2nd), Isosorbide Mononitrate and Levetiracetam (Joint 3rd). We thank panel members and the wider investigator team for their support.siqbal2482@gmail.com