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

Autor: Bedlack R; Duke University Department of Neurology, Durham, NC, USA., Li X; Duke University Department of Neurology, Durham, NC, USA., Evangelista BA; University of North Carolina Department of Neurology, Chapel Hill, NC, USA., Panzetta ME; Duke University Department of Integrative Immunobiology, Durham, NC, USA., Kwan J; Neurodegeneration Disorders Clinic, National Institute of Health, Bethesda, MD, USA., Gittings LM; Department of Translational Neuroscience, Barrow Neurological Institute, Phoenix, AZ, USA., Sattler R; Department of Translational Neuroscience, Barrow Neurological Institute, Phoenix, AZ, USA.
Jazyk: angličtina
Zdroj: Annals of neurology [Ann Neurol] 2024 Nov 06. Date of Electronic Publication: 2024 Nov 06.
DOI: 10.1002/ana.27126
Abstrakt: 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.
(© 2024 The Author(s). Annals of Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association.)
Databáze: MEDLINE
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