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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Veröffentlicht in:Annals of neurology 2025-01, Vol.97 (1), p.15-27
Hauptverfasser: Bedlack, Richard, Li, Xiaoyan, Evangelista, Baggio Angelo, Panzetta, Maria E., Kwan, Justin, Gittings, Lauren M., Sattler, Rita
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Sprache:eng
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Zusammenfassung: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 2025;97:15–27 Eight alternative or off‐label treatments have been identified that all have plausible mechanisms for treating amyotrophic lateral sclerosis and some benefits in small, flawed clinical trials. Created with BioRender.com. [Color figure can be viewed at www.annalsofneurology.org]
ISSN:0364-5134
1531-8249
1531-8249
DOI:10.1002/ana.27126