The Scientific and Therapeutic Rationale for Off-Label Treatments in Amyotrophic Lateral Sclerosis

There are no dramatically effective pharmacological treatments for most patients with amyotrophic lateral sclerosis, a complex disease with multiple underlying mechanisms, such as neuroinflammation, oxidative stress, mitochondrial dysfunction, microbiome alteration, and antiretroviral activity. We s...

Full description

Saved in:
Bibliographic Details
Published in:Annals of neurology 2024-11
Main Authors: Bedlack, Richard, Li, Xiaoyan, Evangelista, Baggio Angelo, Panzetta, Maria E, Kwan, Justin, Gittings, Lauren M, Sattler, Rita
Format: Article
Language:English
Citations: Items that this one cites
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:There are no dramatically effective pharmacological treatments for most patients with amyotrophic lateral sclerosis, a complex disease with multiple underlying mechanisms, such as neuroinflammation, oxidative stress, mitochondrial dysfunction, microbiome alteration, and antiretroviral activity. We sifted through 15 years of reviews by a group called ALSUntangled to identify 8 alternative and off-label treatments that target ≥1 of these mechanisms, and have ≥1 human trial suggesting meaningful benefits. Given the overlapping pathological mechanisms of the highlighted products, we suggest that combinations of these treatments targeting diverse mechanisms might be worthwhile for future amyotrophic lateral sclerosis therapy development. ANN NEUROL 2024.
ISSN:0364-5134
1531-8249
1531-8249
DOI:10.1002/ana.27126