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. 2021 Mar;92(3):295-302.
doi: 10.1136/jnnp-2020-324286. Epub 2020 Nov 12.

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

Nick Cunniffe  1 , Khue Anh Vuong  2 , Debbie Ainslie  3 , David Baker  4 , Judy Beveridge  3 , Sorrel Bickley  2 , Patrick Camilleri  5 , Matthew Craner  6 , Denise Fitzgerald  7 , Alerie G de la Fuente  7 , Gavin Giovannoni  4 , Emma Gray  2 , Lorraine Hazlehurst  3 , Raj Kapoor  8 , Ranjit Kaur  3 , David Kozlowski  3 , Brooke Lumicisi  2 , Don Mahad  9 , Björn Neumann  10 , Alan Palmer  11 , Luca Peruzzotti-Jametti  10 , Stefano Pluchino  10 , Jennifer Robertson  2 , Alan Rothaul  12 , Lyndsey Shellard  3 , Kenneth J Smith  13 , Alastair Wilkins  14 , Anna Williams  15 , Alasdair Coles  10
Affiliations

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

Nick Cunniffe et al. J Neurol Neurosurg Psychiatry. 2021 Mar.

Abstract

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.

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Conflict of interest statement

Competing interests: DB received compensation for consultancy activity from Canbex Therapeutics, Japan Tobacco, Lundbeck, InMune Bio, Merck, Novartis, and Roche in the past 3 years. AC received honoraria and travel support from Genzyme (a Sanofi company) prior to 2017. MC has received honoraria for educational events and/or consultancy from Biogen, Merck, Roche, AbbVie and Novartis. GG has received compensation for serving as a consultant in relation to multiple sclerosis drug development from AbbVie, Actelion, Atara Bio, Biogen, Celgene, EMD Serono, Japanese Tobacco, Sanofi-Genzyme, Genentech, GlaxoSmithKline, GW Pharma, Merck KGa, Novartis, Roche and Teva. LH holds a small number of GSK shares as part of her renumeration when she was an employee, which she left 4 years ago. DM received consultancy fees from Biogen, MedDay and SanofiGenzyme and Novartis. BN holds a patent regarding the treatment of demyelinating diseases including metformin: WO2019/206419 A1, Treatment for demyelinating disease. SP is cofounder, CSO and shareholder (>5%) of CITC Ltd and iSTEM Therapeutics, and cofounder and non-executive director at Asitia Therapeutics. LP-J is Head of Research at iSTEM Therapeutics. AW receives research support from Roche not associated with drug development or use.

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