RT Journal Article SR Electronic T1 Systematic approach to selecting licensed drugs for repurposing in the treatment of progressive multiple sclerosis JF Journal of Neurology, Neurosurgery & Psychiatry JO J Neurol Neurosurg Psychiatry FD BMJ Publishing Group Ltd SP 295 OP 302 DO 10.1136/jnnp-2020-324286 VO 92 IS 3 A1 Nick Cunniffe A1 Khue Anh Vuong A1 Debbie Ainslie A1 David Baker A1 Judy Beveridge A1 Sorrel Bickley A1 Patrick Camilleri A1 Matthew Craner A1 Denise Fitzgerald A1 Alerie G de la Fuente A1 Gavin Giovannoni A1 Emma Gray A1 Lorraine Hazlehurst A1 Raj Kapoor A1 Ranjit Kaur A1 David Kozlowski A1 Brooke Lumicisi A1 Don Mahad A1 Björn Neumann A1 Alan Palmer A1 Luca Peruzzotti-Jametti A1 Stefano Pluchino A1 Jennifer Robertson A1 Alan Rothaul A1 Lyndsey Shellard A1 Kenneth J Smith A1 Alastair Wilkins A1 Anna Williams A1 Alasdair Coles YR 2021 UL http://jnnp.bmj.com/content/92/3/295.abstract AB 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.