TY - JOUR
T1 - Remyelination trials
T2 - are we expecting the unexpected?
AU - Klistorner, Alexander
AU - Barnett, Michael
N1 - Copyright the Author(s) 2021. Version archived for private and non-commercial use with the permission of the author/s and according to publisher conditions. For further rights please contact the publisher.
PY - 2021/11/1
Y1 - 2021/11/1
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85113872862&partnerID=8YFLogxK
U2 - 10.1212/NXI.0000000000001066
DO - 10.1212/NXI.0000000000001066
M3 - Review article
C2 - 34376551
AN - SCOPUS:85113872862
SN - 2332-7812
VL - 8
SP - 1
EP - 5
JO - Neurology: Neuroimmunology and Neuroinflammation
JF - Neurology: Neuroimmunology and Neuroinflammation
IS - 6
M1 - e1066
ER -