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Emerging Drugs for the Treatment of Amyotrophic Lateral Sclerosis: A Focus on Recent Phase 2 Trials.
- Source :
-
Expert opinion on emerging drugs [Expert Opin Emerg Drugs] 2020 Jun; Vol. 25 (2), pp. 145-164. Date of Electronic Publication: 2020 May 27. - Publication Year :
- 2020
-
Abstract
- Introduction: Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disease involving both upper and lower motor neurons and resulting in increasing disability and death 3-5 years after onset of symptoms. Over 40 large clinical trials for ALS have been negative, except for Riluzole that offers a modest survival benefit, and Edaravone that modestly reduces disease progression in patients with specific characteristics. Thus, the discovery of efficient disease modifying therapy is an urgent need.<br />Areas Covered: Although the cause of ALS remains unclear, many studies have demonstrated that neuroinflammation, proteinopathies, glutamate-induced excitotoxicity, microglial activation, oxidative stress, and mitochondrial dysfunction may play a key role in the pathogenesis. This review highlights recent discoveries relating to these diverse mechanisms and their implications for the development of therapy. Ongoing phase 2 clinical trials aimed to interfere with these pathophysiological mechanisms are discussed.<br />Expert Opinion: This review describes the challenges that the discovery of an efficient drug therapy faces and how these issues may be addressed. With the continuous advances coming from basic research, we provided possible suggestions that may be considered to improve performance of clinical trials and turn ALS research into a 'fertile ground' for drug development for this devastating disease.
- Subjects :
- Amyotrophic Lateral Sclerosis physiopathology
Animals
Disease Progression
Drug Discovery
Edaravone administration & dosage
Edaravone pharmacology
Humans
Neuroprotective Agents pharmacology
Oxidative Stress drug effects
Riluzole administration & dosage
Riluzole pharmacology
Amyotrophic Lateral Sclerosis drug therapy
Drug Development
Neuroprotective Agents administration & dosage
Subjects
Details
- Language :
- English
- ISSN :
- 1744-7623
- Volume :
- 25
- Issue :
- 2
- Database :
- MEDLINE
- Journal :
- Expert opinion on emerging drugs
- Publication Type :
- Academic Journal
- Accession number :
- 32456491
- Full Text :
- https://doi.org/10.1080/14728214.2020.1769067