514 - Assessing the Efficacy of Amyotrophic Lateral Sclerosis Drugs in Slowing Disease Progression: A Literature Review

A PubMed search was conducted, and relevant articles from the past 20 years were included in the final manuscript. A narrative review was conducted to explore the efficacy of current FDA-approved pharmacotherapy treatments for ALS.

Results:

Qalsody (tofersen), represents a groundbreaking treatment targeting ALS associated with mutations in the superoxide dismutase 1 (SOD1) gene. Relyvrio is another promising drug that combines sodium phenylbutyrate and taurursodiol to intervene in nerve cell death processes by intercepting stress signals within cells. Radicava (edaravone) marked the first novel ALS treatment in over two decades, underscoring its importance in disease management. Rilutek (riluzole), the first drug approved by the FDA to treat ALS, inhibits glutamate release and extends patients’ lives by approximately three months. Tiglutik is a new thickened liquid form of riluzole that provides an alternative to the traditional oral pill, thereby addressing potential issues related to tablet administration. Exservan introduces an oral film formulation of riluzole. It is specifically designed for ALS patients experiencing severe dysphagia. Nuedexta is another promising drug that combines dextromethorphan hydrobromide (HBr) and quinidine sulfate to manage pseudobulbar affect (PBA).

Conclusions: Despite these advancements in pharmaceutical interventions, finding a definitive cure for ALS remains a significant challenge. Continuous investigation into ALS pathophysiology and therapeutic avenues remains imperative, necessitating further research collaborations and innovative approaches to unravel the complex mechanisms underlying this debilitating condition.