Status of ALS treatment, insights into therapeutic challenges and dilemmas
Open accessAbstract
Amyotrophic lateral sclerosis (ALS) is an extremely heterogeneous disease of motor neurons that eventually leads to death. Despite impressive advances in understanding the genetic, molecular, and pathological mechanisms of the disease, the only drug approved to date by both the FDA and EMA is riluzole, with a modest effect on survival. In this opinion view paper, we will discuss how to address some challenges for drug development in ALS at the conceptual, technological, and methodological levels. In addition, socioeconomic and ethical issues related to the legitimate need of patients to benefit quickly from new treatments will also be addressed. In conclusion, this brief review takes a more optimistic view, given the recent approval of two new drugs in some countries and the development of targeted gene therapies.
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Edaravone has been approved by the FDA for oral use and possesses potent antioxidant properties that mitigate oxidative stress by eliminating oxidized lipids and hydroxyl radicals.
“Another drug, edaravone, has also been approved by the FDA for oral use (9) and possesses potent antioxidant properties that mitigate the effects of oxidative stress by eliminating oxidized lipids and hydroxyl radicals, which may be a key factor in the pathogenesis and development of ALS.”
From Modulation of the gut–brain axis via the gut microbiota: a new era in treatment of amyotrophic lateral sclerosis (2023) · cites this paper as [9]
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References 129 · Cited by 28
Research works printed but not linked: 2
- [32]Astrazeneca Update on CHAMPION-ALS Phase III Trial of Ultomiris in Amyotrophic Lateral Sclerosis Available online: https://www.astrazeneca.com/media-centre/press-releases/2021/update-on-ultomiris-phase-iii-als-trial.html (accessed on 28 February 2022)
- [33]Hospital M.G. HEALEY ALS Platform Trial Update: Zilucoplan Arm Stopped Early for Futility Available online: https://www.massgeneral.org/news/press-release/healey-als-platform-trial-update-ilucoplan-arm-stopped-early-for-futility (accessed on 3 March 2022)
- Authors
- Khamaysa, M., & Pradat, P.-F.
- Affiliations
- Centre National de la Recherche Scientifique (FR)
- Corresponding author
- Khamaysa, Mohammed
- Pradat, Pierre-François
- Venue
- Journal of Personalized Medicine
- Publisher
- MDPI AG
- Published
- 2022-09-28
- Volume
- 12
- Issue
- 10
- Pages
- 1601
- ISSN
- 2075-4426 (ISSN-L)
- PMC
- PMC9605458
- OpenAlex
- W4297478874
- Cited by
- 28
- References
- 129
- Citations by year
- 2026: 3 · 2025: 5 · 2024: 13 · 2023: 6 · 2022: 1
- FWCI
- 0.90
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Journal of Personalized Medicine (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- Ulster University Research Portal (Ulster University) (submittedVersion, cc-by)
- HAL (submittedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion)
- Funders
- Strong
- Association pour la Recherche sur la Sclérose Latérale Amyotrophique et autres Maladies du Motoneurone
- Topics
- Health Sciences › Medicine › Neurology › Amyotrophic Lateral Sclerosis Research
- Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
- Health Sciences › Medicine › Physiology › Alzheimer's disease research and treatments
- Keywords
- amyotrophic lateral sclerosis, riluzole, motor neuron disease, gene therapy, treatment challenges, socio-economic problems, ethical considerations, drug approval
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