Cargando…

Current State and Future Directions in the Therapy of ALS

Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder affecting upper and lower motor neurons, with death resulting mainly from respiratory failure three to five years after symptom onset. As the exact underlying causative pathological pathway is unclear and potenti...

Descripción completa

Detalles Bibliográficos
Autores principales: Tzeplaeff, Laura, Wilfling, Sibylle, Requardt, Maria Viktoria, Herdick, Meret
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10252394/
https://www.ncbi.nlm.nih.gov/pubmed/37296644
http://dx.doi.org/10.3390/cells12111523
_version_ 1785056160736346112
author Tzeplaeff, Laura
Wilfling, Sibylle
Requardt, Maria Viktoria
Herdick, Meret
author_facet Tzeplaeff, Laura
Wilfling, Sibylle
Requardt, Maria Viktoria
Herdick, Meret
author_sort Tzeplaeff, Laura
collection PubMed
description Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder affecting upper and lower motor neurons, with death resulting mainly from respiratory failure three to five years after symptom onset. As the exact underlying causative pathological pathway is unclear and potentially diverse, finding a suitable therapy to slow down or possibly stop disease progression remains challenging. Varying by country Riluzole, Edaravone, and Sodium phenylbutyrate/Taurursodiol are the only drugs currently approved in ALS treatment for their moderate effect on disease progression. Even though curative treatment options, able to prevent or stop disease progression, are still unknown, recent breakthroughs, especially in the field of targeting genetic disease forms, raise hope for improved care and therapy for ALS patients. In this review, we aim to summarize the current state of ALS therapy, including medication as well as supportive therapy, and discuss the ongoing developments and prospects in the field. Furthermore, we highlight the rationale behind the intense research on biomarkers and genetic testing as a feasible way to improve the classification of ALS patients towards personalized medicine.
format Online
Article
Text
id pubmed-10252394
institution National Center for Biotechnology Information
language English
publishDate 2023
publisher MDPI
record_format MEDLINE/PubMed
spelling pubmed-102523942023-06-10 Current State and Future Directions in the Therapy of ALS Tzeplaeff, Laura Wilfling, Sibylle Requardt, Maria Viktoria Herdick, Meret Cells Review Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder affecting upper and lower motor neurons, with death resulting mainly from respiratory failure three to five years after symptom onset. As the exact underlying causative pathological pathway is unclear and potentially diverse, finding a suitable therapy to slow down or possibly stop disease progression remains challenging. Varying by country Riluzole, Edaravone, and Sodium phenylbutyrate/Taurursodiol are the only drugs currently approved in ALS treatment for their moderate effect on disease progression. Even though curative treatment options, able to prevent or stop disease progression, are still unknown, recent breakthroughs, especially in the field of targeting genetic disease forms, raise hope for improved care and therapy for ALS patients. In this review, we aim to summarize the current state of ALS therapy, including medication as well as supportive therapy, and discuss the ongoing developments and prospects in the field. Furthermore, we highlight the rationale behind the intense research on biomarkers and genetic testing as a feasible way to improve the classification of ALS patients towards personalized medicine. MDPI 2023-05-31 /pmc/articles/PMC10252394/ /pubmed/37296644 http://dx.doi.org/10.3390/cells12111523 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Tzeplaeff, Laura
Wilfling, Sibylle
Requardt, Maria Viktoria
Herdick, Meret
Current State and Future Directions in the Therapy of ALS
title Current State and Future Directions in the Therapy of ALS
title_full Current State and Future Directions in the Therapy of ALS
title_fullStr Current State and Future Directions in the Therapy of ALS
title_full_unstemmed Current State and Future Directions in the Therapy of ALS
title_short Current State and Future Directions in the Therapy of ALS
title_sort current state and future directions in the therapy of als
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10252394/
https://www.ncbi.nlm.nih.gov/pubmed/37296644
http://dx.doi.org/10.3390/cells12111523
work_keys_str_mv AT tzeplaefflaura currentstateandfuturedirectionsinthetherapyofals
AT wilflingsibylle currentstateandfuturedirectionsinthetherapyofals
AT requardtmariaviktoria currentstateandfuturedirectionsinthetherapyofals
AT herdickmeret currentstateandfuturedirectionsinthetherapyofals