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Systematic approach to selecting licensed drugs for repurposing in the treatment of progressive multiple sclerosis.

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Vuong, Khue Anh 
Ainslie, Debbie 
Beveridge, Judy 


OBJECTIVE: To establish a rigorous, expert-led, evidence-based approach to the evaluation of licensed drugs for repurposing and testing in clinical trials of people with progressive multiple sclerosis (MS). METHODS: We long-listed licensed drugs with evidence of human safety, blood-brain barrier penetrance and demonstrable efficacy in at least one animal model, or mechanistic target, agreed by a panel of experts and people with MS to be relevant to the pathogenesis of progression. We systematically reviewed the preclinical and clinical literature for each compound, condensed this into a database of summary documents and short-listed drugs by scoring each one of them. Drugs were evaluated for immediate use in a clinical trial, and our selection was scrutinised by a final independent expert review. RESULTS: From a short list of 55 treatments, we recommended four treatments for immediate testing in progressive MS: R-α-lipoic acid, metformin, the combination treatment of R-α-lipoic acid and metformin, and niacin. We also prioritised clemastine, lamotrigine, oxcarbazepine, nimodipine and flunarizine. CONCLUSIONS: We report a standardised approach for the identification of candidate drugs for repurposing in the treatment of progressive MS.



Animals, Drug Evaluation, Drug Repositioning, Humans, Multiple Sclerosis, Chronic Progressive

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J Neurol Neurosurg Psychiatry

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JB received expense payments from Novartis for speaking as patient representative during Siponimod licensing. AJC receives funding from the MRC and MS Society UK. DF is funded by the Wellcome and BBSRC, and has a project with Sangamo. A.G. de la Fuente has been supported by the ECTRIMS postdoctoral fellowship during this period. GG declares current research funding from Merck KGa (CLAD-B study), Roche (ORATORIO-HAND study) and Takeda (SIZOMUS Study). DM received funding previously from Biogen, MedDay and SanofiGenzyme. BN received funding from the Cambridge Centre for Myelin Repair, funded by MS Society UK. SP declares current funding from Italian and US Multiple Sclerosis Societies. LP has been supported by a senior research fellowship FISM - Fondazione Italiana Sclerosi Multipla - cod. 2017/B/5 and financed or co financed with the ‘5 per mille' public funding, by the Isaac Newton Trust RG 97440 and the Addenbrooke’s Charitable Trust RG 97519. KS declares current funding from Fondation Leducq, Multiple Sclerosis Society, Rosetrees Trust. A. Wilkins received a research grant from Sanofy (2018). A. Williams declares funding from MS Society UK, Roche, MRC, Lifearc.