Role of Edaravone as a Treatment Option for Patients with Amyotrophic Lateral Sclerosis

Abstract

Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive and fatal neurodegenerative disease that leads to a loss of muscle control due to nerve cells being affected in the brain and spinal cord. Some of the common clinical presentations of ALS include weakness of muscles, changes in behavior, dysfunction in speech, and cognitive difficulties. The cause of ALS is uncertain, but through several studies, it is known that mutations in SOD1 or C9orf72 genes could play a role as a factor of ALS. In addition, studies indicate that an excessive amount of free radicals, the reactive oxygen species (ROS), leads to neuronal damage by the peroxidation of unsaturated fatty acids in the neuronal cells. Edaravone, the newly approved antioxidant drug for ALS, halts the progression of ALS in the early stages through its cytoprotective effect and protects the nerves by reducing ROS. In this review, different aspects of ALS will be discussed, including its pathology, genetic aspect, and diagnosis. This review also focuses on edaravone as a treatment option for ALS, its mechanism of action, and its pharmacological properties. Clinical trials and adverse effects of edaravone and care for ALS patient are also discussed.

Improved Long-Term Survival with Edaravone Therapy in Patients with Amyotrophic Lateral Sclerosis: A Retrospective Single-Center Study in Japan

“…These mechanisms demonstrate that edaravone is effective in delaying the progression of symptoms in animal models of ALS [ 2 ] indicating its clinical application. The approval of edaravone for marketing and manufacturing in Japan in 2015 was followed by its approval for the treatment of ALS in the USA by the Food and Drug Administration in 2017 [ 4 ]. A phase III randomized double-blind study was performed to determine the safety and efficacy of 60 mg/day edaravone administered intravenously for 2 weeks each month in patients with early-stage ALS [ 5 ].…”

“…In addition, Japan was among the first countries to approve edaravone for the treatment of ALS in 2015. Subsequently, the Food and Drug Administration approved edaravone for the treatment of ALS in 2017 [ 4 ]. A placebo-controlled phase III trial of edaravone in patients with mild ALS demonstrated that, compared with placebo, edaravone led to a significantly smaller decline in revised ALS functional rating scale (ALSFRS-R) scores over 24 weeks [ 5 ].…”

The Skeletal Muscle Emerges as a New Disease Target in Amyotrophic Lateral Sclerosis

“…Despite being the most common degenerative MN disease, ALS is classified as a rare disease based on its low prevalence due to its high mortality; the survival rate is relatively short, between 2 and 5 years on average [ 3 ], placing ALS as one of the most devastating diseases among all deadly disorders. Current treatments, such as the glutamate antagonist Riluzole, or the free radical scavenger Edaravone (only approved in few countries), slow down disease progression but are unable to reverse nerve damage or muscle weakness [ 4 , 5 ].…”

Mesenchymal Stem Cells in Treatment of Spinal Cord Injury and Amyotrophic Lateral Sclerosis

“…Poor prognosis and symptomatic treatment are so far the only prospect for patients. The pharmaceutical treatments used in all patients include glutamate inhibition with riluzole ( Bensimon et al, 1994 ), which only extends survival by about 3 months, or free radical scavenger edaravone ( Cho and Shukla, 2020 ). Both of these drugs only delay the symptomatic and pathological progression of ALS.…”