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Why is ALS so Difficult to Treat?

Published online by Cambridge University Press:  23 September 2014

John Turnbull
John Turnbull*
Affiliation:
Department of Medicine, McMaster University, Hamilton, Ontario, Canada
*
Andrew Bruce Douglas Chair of Neurology, Department of Medicine, McMaster University, 1200 Main St W, Hamilton Ontario, L8N 3Z5, Canada. email: turnbull@mcmaster.ca
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Abstract

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Amyotrophic lateral sclerosis (ALS) is proving intractable. Difficulties in pre-clinical studies contribute in small measure to this futility, but the chief reason for failure is an inadequate understanding of disease pathogenesis. Many acquired and inherited processes have been advanced as potential causes of ALS but, while they may predispose to disease, it seems increasingly likely that none leads directly to ALS. Rather, two recent overlapping considerations, both involving aberrant protein homeostasis, may provide a better explanation for a common disease phenotype and a common terminal pathogenesis. If so, therapeutic approaches will need to be altered and carefully nuanced, since protein homeostasis is essential and highly conserved. Nonetheless, these considerations provide new optimism in a difficult disease which has hitherto defied treatment.

Type
Review Article
Information
Canadian Journal of Neurological Sciences , Volume 41 , Issue 2 , March 2014 , pp. 144 - 155
Copyright
Copyright © The Canadian Journal of Neurological 2014

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