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...

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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
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container_title Annals of neurology
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creator Bedlack, Richard
Li, Xiaoyan
Evangelista, Baggio Angelo
Panzetta, Maria E
Kwan, Justin
Gittings, Lauren M
Sattler, Rita
description 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.
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title The Scientific and Therapeutic Rationale for Off-Label Treatments in Amyotrophic Lateral Sclerosis
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