Special Issue "Edaravone for the Treatment of Amyotrophic Lateral Sclerosis"

A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Medicinal Chemistry".

Deadline for manuscript submissions: 28 February 2022.

Special Issue Editor

Dr. Hiide Yoshino
E-Mail Website
Guest Editor
Yoshino Neurology Clinic, Ichikawa, Japan
Interests: amyotrophic lateral sclerosis (ALS); edaravone; free radicals; oxidative stress

Special Issue Information

Dear Colleagues,

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease. Most patients die within 3–5 years from onset. All ALS patients are desperate for new treatment. It took almost 20 years after riluzole approval in 1995 until edaravone was approved in Japan in 2015 and in US in 2017. Although the first phase 3 trial collecting a wide variety of ALS patients failed to show the efficacy of edaravone, the second phase 3 trial succeeded in proving its efficacy in inhibiting the deterioration of motor functions by approximately 33% compared to placebo. The results of these clinical trials tell us early diagnosis is critically important for the benefit of ALS patients to prolong better motor functions for longer. Development of biomarkers for early diagnosis is critically important. At present, edaravone is limited to intravenous administration. The oral formula of edaravone is under clinical trial for 12 months of safety assessment. In order to investigate more efficacious edaravone treatment options, a double-blind study is being launched to show the efficacy of everyday administration compared to current protocol with 10 days in 4 weeks administration. As free radical scavenging has been proven to be a key treatment strategy, further development of new therapeutic agents that target free radicals could lead to better outcomes for ALS treatment.

Dr. Hiide Yoshino
Guest Editor

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Keywords

  • ALS
  • diagnosis
  • treatment
  • edaravone
  • biomarker
  • free radicals
  • oxidative stress
  • clinical trial

Published Papers (2 papers)

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Research

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Article
Improved Long-Term Survival with Edaravone Therapy in Patients with Amyotrophic Lateral Sclerosis: A Retrospective Single-Center Study in Japan
Pharmaceuticals 2021, 14(8), 705; https://0-doi-org.brum.beds.ac.uk/10.3390/ph14080705 - 21 Jul 2021
Viewed by 294
Abstract
Reports on the long-term survival effect of edaravone, which was approved for the treatment of amyotrophic lateral sclerosis (ALS) in 2015 in Japan, are rare. Herein, we report our retrospective analysis of 45 consecutive patients with ALS who initially visited our hospital between [...] Read more.
Reports on the long-term survival effect of edaravone, which was approved for the treatment of amyotrophic lateral sclerosis (ALS) in 2015 in Japan, are rare. Herein, we report our retrospective analysis of 45 consecutive patients with ALS who initially visited our hospital between 2013 and 2018. Of these, 22 patients were treated with edaravone for an average duration of 26.6 (range, 2–64) months, whereas the remaining patients were not treated with edaravone and comprised the control group. There were no differences in baseline demographics between the two groups. The primary endpoint was tracheostomy positive-pressure ventilation (TPPV) or death, and the follow-up period ended in December 2020. The survival rate was significantly better in the edaravone group than in the control group based on the Kaplan–Meier analysis, which revealed that the median survival durations were 49 (9–88) and 25 (8–41) months in the edaravone and control groups, respectively (p = 0.001, log-rank test). There were no serious edaravone-associated adverse effects during the study period. Overall, the findings of this single-center retrospective study suggest that edaravone might prolong survival in patients with ALS. Full article
(This article belongs to the Special Issue Edaravone for the Treatment of Amyotrophic Lateral Sclerosis)
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Review

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Review
Role of Edaravone as a Treatment Option for Patients with Amyotrophic Lateral Sclerosis
Pharmaceuticals 2021, 14(1), 29; https://doi.org/10.3390/ph14010029 - 31 Dec 2020
Cited by 4 | Viewed by 1011
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 [...] Read more.
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. Full article
(This article belongs to the Special Issue Edaravone for the Treatment of Amyotrophic Lateral Sclerosis)
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