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ALS Clinical Trials Review: 20 Years of Failure. Are We Any Closer to Registering a New Treatment?
Open accessAbstract
Amyotrophic lateral sclerosis (ALS) is a devastating condition with an estimated mortality of 30,000 patients a year worldwide. The median reported survival time since onset ranges from 24 to 48 months. Riluzole is the only currently approved mildly efficacious treatment. Riluzole received marketing authorization in 1995 in the USA and in 1996 in Europe. In the years that followed, over 60 molecules have been investigated as a possible treatment for ALS. Despite significant research efforts, the overwhelming majority of human clinical trials (CTs) have failed to demonstrate clinical efficacy. In the past year, oral masitinib and intravenous edaravone have emerged as promising new therapeutics with claimed efficacy in CTs in ALS patients. Given their advanced phase of clinical development one may consider these drugs as the most likely near-term additions to the therapeutic arsenal available for patients with ALS. In terms of patient inclusion, CT with masitinib recruited a wider, more representative, less restrictive patient population in comparison to the only successful edaravone CT (edaravone eligibility criteria represents only 18% of masitinib study patients). The present manuscript reviews >50 CTs conducted in the last 20 years since riluzole was first approved. A special emphasis is put on the analysis of existing evidence in support of the clinical efficacy of edaravone and masitinib and the possible implications of an eventual marketing authorisation in the treatment of ALS.
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“Multiple small molecules with anti-inflammatory activity, including minocycline, masitinib, NP001, and celecoxib, have been tested for the treatment of ALS. Although most have failed, masitinib was found to be effective in the preceding clinical trials, and a Phase III clinical trial testing masitinib in ALS patients is ongoing.577”
From Role of neuroinflammation in neurodegeneration development (2023) · cites this paper as [577]
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“Probiotics are considered to be dietary factors that can influence the human intestinal microbiota, which can affect the composition and structure of the intestinal flora; which, in turn, plays a role in protecting the intestinal epithelial barrier, inhibiting pathogen production, and regulating the immune system (3).”
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 [3]
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“Through use of probiotics, the number of SCFA-producing microorganisms in the intestine can be increased (3).”
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 [3]
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Research works printed but not linked: 5
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Ageing as a risk factor for neurodegenerative disease
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“Although most have failed, masitinib was found to be effective in the preceding clinical trials, and a Phase III clinical trial testing masitinib in ALS patients is ongoing.(577) dl-3-nButylphthalide (dl-NBP), a synthetic compound based on l-3-nButylphthalide that is isolated from seeds of Apium graveolens, has been f…”
BackgroundMolecular Mechanisms of TDP-43 Misfolding and Pathology in Amyotrophic Lateral Sclerosis
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ALS Genetics, Mechanisms, and Therapeutics: Where Are We Now?
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Metadata
- Authors
- Petrov, D., Mansfield, C., Moussy, A., & Hermine, O.
- Affiliations
- AB Science (France) (FR)
- Hôpital Necker-Enfants Malades (FR)
- Corresponding author
- Hermine, Olivier
- Venue
- Frontiers in Aging Neuroscience
- Publisher
- Frontiers Media SA
- Published
- 2017-03-22
- Volume
- 9
- Pages
- 68-68
- ISSN
- 1663-4365 (ISSN-L)
- PMC
- PMC5360725
- OpenAlex
- W2597399186
- Cited by
- 451
- References
- 108
- Citations by year
- 2026: 28 · 2025: 41 · 2024: 45 · 2023: 46 · 2022: 65 · 2021: 63 · 2020: 53 · 2019: 52 · 2018: 50 · 2017: 8
- FWCI
- 5.94
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- cc-by
- Locations
- Frontiers in Aging Neuroscience (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- Funders
- Agence Nationale de la Recherche (ANR-11-LABX-0051)
- Topics
- Health Sciences › Medicine › Clinical Neurology › Amyotrophic Lateral Sclerosis Research
- Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Alzheimer's disease research and treatments
- Health Sciences › Medicine › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
- Keywords
- amyotrophic lateral sclerosis, clinical trial failure, riluzole, edaravone, masitinib, drug development, clinical efficacy, patient selection criteria, drug approval, neurodegenerative disease therapeutics
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
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