Risk factors and emerging therapies in amyotrophic lateral sclerosis
Open accessAbstract
Amyotrophic lateral sclerosis (ALS) is a fatal progressive neurodegenerative disease characterized by a permanent degeneration of both upper and lower motor neurons. Many different genes and pathophysiological processes contribute to this disease, however its exact cause remains unclear. Therefore, it is necessary to understand this heterogeneity to find effective treatments. In this review, we focus on selected environmental and genetic risk factors predisposing to ALS and highlight emerging treatments in ALS therapy. Of numerous defective genes associated with ALS, we focus on four principal genes that have been identified as definite causes of ALS: the SOD1 gene, C9orf72, TDP-43, as well as the recently identified TBK1. We also provide up-to-date information on selected environmental factors that have historically been considered as key players in ALS development and pathogenesis. In parallel to our survey of known risk factors, we also discuss emerging ALS stem cell therapies and experimental medicines currently undergoing phase II and III clinical trials.
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Riluzole is the most widely used disease-modifying drug for ALS, but its effectiveness is limited.
“two recognized therapeutic agents have been classified as disease-modifying drugs, with riluzole being the most widely used (6). Despite its ability to slow the progression of ALS, its effectiveness is limited.”
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 [6]
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- Authors
- Nowicka, N., Juranek, J., Juranek, J. K., & Wojtkiewicz, J.
- Affiliations
- University of Warmia and Mazury in Olsztyn (PL)
- Institute of Psychology (PL)
- Corresponding author
- Nowicka, Natalia
- Wojtkiewicz, Joanna
- Venue
- International Journal of Molecular Sciences
- Publisher
- MDPI AG
- Published
- 2019-05-28
- Volume
- 20
- Issue
- 11
- Pages
- 2616
- ISSN
- 1422-0067 (ISSN-L)
- PMC
- PMC6600314
- OpenAlex
- W2947955929
- Cited by
- 106
- References
- 154
- Citations by year
- 2026: 11 · 2025: 10 · 2024: 11 · 2023: 22 · 2022: 17 · 2021: 12 · 2020: 20 · 2019: 3
- FWCI
- 1.74
- Type
- journal article
- Language
- en
- Open access
- gold
- dx.doi.org
- Licence
- Locations
- International Journal of Molecular Sciences (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion)
- Funders
- Narodowym Centrum Nauki (OPUS/2017/25/B/NZ4/00435)
- Topics
- Health Sciences › Medicine › Neurology › Amyotrophic Lateral Sclerosis Research
- Health Sciences › Medicine › Physiology › Alzheimer's disease research and treatments
- Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Hereditary Neurological Disorders
- Keywords
- amyotrophic lateral sclerosis, risk factors, genetic risk factors, environmental risk factors, SOD1, C9orf72, TDP-43, TBK1, motor neuron degeneration, stem cell therapy, experimental treatment, clinical trials
- MeSH terms
- Superoxide Dismutase-1, C9orf72 Protein, Amyotrophic Lateral Sclerosis, Animals, DNA-Binding Proteins, Humans, Genetic Therapy, Genetic Predisposition to Disease, Molecular Targeted Therapy
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