Update on ALS Treatment

• Published in: Current treatment options in neurology 2023-07, Vol.25 (7), p.199-212
• Main Authors: Priyadarshini, Shubadra, Ajroud-Driss, Senda
• Format: Article
• Language: English
• Subjects: Critical Care Medicine; Diabetes; Family Medicine; General Practice; Intensive; Internal Medicine; Medicine; Medicine & Public Health; Neurology; Ophthalmology; Topical Collection on Neuromuscular Disorders
• ISSN: 1092-8480; 1534-3138
• DOI: 10.1007/s11940-023-00757-4

Purpose of Review While amyotrophic lateral sclerosis (ALS) remains a progressive fatal diagnosis, there have been numerous advances in the last several years, both in disease-modifying and symptomatic treatment. This review aims to provide a comprehensive and updated review of the literature of current treatment options for ALS. Recent Findings We will discuss the proposed mechanisms of action, evidence for efficacy, and safety profiles for the four current Food and Drug Administration (FDA)–approved disease-modifying treatments: riluzole, edaravone, AMX0035 (combination of sodium phenylbutyrate and taurursodiol) and tofersen. Additionally, we will review several therapies that are under active investigation for the more common genetic forms of ALS. Finally, we will discuss options for symptomatic treatment, including a review of Nuedexta (combination of dextromethorphan hydrobromide and quinidine sulfate) approved for pseudobulbar affect, but recent evidence is suggesting that it also improves bulbar function. Summary There are four FDA-approved disease-modifying treatments for ALS, which likely confer a modest benefit in survival with good safety profile and tolerability, through different mechanisms of action. More post-marketing and population studies will be needed to assess the overall efficacy of each medication and potential combinations of each.