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November 2021; 8 (6) Views & ReviewsOpen Access

Remyelination Trials

Are We Expecting the Unexpected?

Alexandr Klistorner, Michael Barnett
First published August 10, 2021, DOI: https://doi.org/10.1212/NXI.0000000000001066
Alexandr Klistorner
From the Sydney University (A.K., M.B.); Maquarie University (A.K.); and Sydney Neuroimaging Analysis Center (M.B.), Australia.
PhD
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Michael Barnett
From the Sydney University (A.K., M.B.); Maquarie University (A.K.); and Sydney Neuroimaging Analysis Center (M.B.), Australia.
PhD
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  • For correspondence: michael@snac.com.au
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Citation
Remyelination Trials
Are We Expecting the Unexpected?
Alexandr Klistorner, Michael Barnett
Neurol Neuroimmunol Neuroinflamm Nov 2021, 8 (6) e1066; DOI: 10.1212/NXI.0000000000001066

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Abstract

Neuroaxonal loss is believed to underpin the progressive disability that characterizes multiple sclerosis (MS). While focal inflammatory demyelination is a principal cause of acute axonal transection and subsequent axonal degeneration, the gradual attrition of permanently demyelinated axons may also contribute to tissue damage, particularly in the progressive phase of the disease. Therefore, remyelination is considered a putative neuroprotective strategy. In this article, we review the potential pitfalls of remyelination trials, provide a framework for their appropriate design and temper the expectations, at times unrealistic, of researchers, regulators and the pharmaceutical industry.

Glossary

DMT=
disease-modifying therapy;
FIDID=
Feline Irradiated Diet-Induced Demyelination;
MS=
multiple sclerosis;
PMD=
Pelizaeus Merzbacher Disease

Footnotes

  • The Article Processing Charge was funded by the authors.

  • Go to Neurology.org/NN for full disclosures. Funding information is provided at the end of the article.

  • Received March 20, 2021.
  • Accepted in final form June 14, 2021.
  • Copyright © 2021 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology.

This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License 4.0 (CC BY-NC-ND), which permits downloading and sharing the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal.

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