Amyotrophic lateral sclerosis: an update on treatments from clinical trials

Wynford-Thomas, Ray and Robertson, Neil P. ORCID: https://orcid.org/0000-0002-5409-4909 2023. Amyotrophic lateral sclerosis: an update on treatments from clinical trials. Journal of Neurology 270 (2) , pp. 1187-1189. 10.1007/s00415-022-11553-6

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Abstract

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease resulting in loss of motor neurons, weakness, and death commonly due to respiratory failure, with a median survival of around 3–5 years. Despite considerable advances in the understanding of aetiology and pathophysiology, interventions that modify the natural disease course remain limited. At present available treatments can minimally prolong survival (riluzole) or slow functional decline (edaravone) and non-invasive ventilation (NIV) can prolong survival and improve quality of life in suitable patients, but tolerability can be a limiting factor. As a result, there remains a clear need for novel therapeutic approaches in ALS. In this month’s journal club, we review three clinical trials evaluating new treatments for ALS; tofersen, levosimendan and combined regulatory T-Lymphocyte/IL-2 therapy.

Item Type:	Article
Date Type:	Publication
Status:	Published
Schools:	Medicine
Additional Information:	License information from Publisher: LICENSE 1: URL: http://creativecommons.org/licenses/by/4.0/, Type: open-access
Publisher:	Springer
ISSN:	0340-5354
Date of First Compliant Deposit:	31 January 2023
Date of Acceptance:	28 December 2022
Last Modified:	03 May 2023 08:07
URI:	https://orca.cardiff.ac.uk/id/eprint/156392

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