Biofluid Biomarkers in the Prognosis of Amyotrophic Lateral Sclerosis: Recent Developments and Therapeutic Applications

Author:

Sanchez-Tejerina Daniel1234ORCID,Llaurado Arnau123ORCID,Sotoca Javier123,Lopez-Diego Veronica123,Vidal Taboada Jose M.24ORCID,Salvado Maria123,Juntas-Morales Raul1234

Affiliation:

1. Neuromuscular Diseases Unit, Neurology Department, Vall d’Hebron Hospital Universitari, Vall d’Hebron Barcelona Hospital Campus, 08035 Barcelona, Spain

2. Peripheral Nervous System Group, Vall d’Hebron Research Institut (VHIR), Vall d’Hebron Barcelona Hospital Campus, 08035 Barcelona, Spain

3. European Reference Network on Rare Neuromuscular Diseases (ERN EURO-NMD), Vall d’Hebron Barcelona Hospital Campus, 08035 Barcelona, Spain

4. Medicine Department, Universitat Autónoma de Barcelona, 08035 Barcelon, Spain

Abstract

Amyotrophic lateral sclerosis is a neurodegenerative disease characterized by the degeneration of motor neurons for which effective therapies are lacking. One of the most explored areas of research in ALS is the discovery and validation of biomarkers that can be applied to clinical practice and incorporated into the development of innovative therapies. The study of biomarkers requires an adequate theoretical and operational framework, highlighting the “fit-for-purpose” concept and distinguishing different types of biomarkers based on common terminology. In this review, we aim to discuss the current status of fluid-based prognostic and predictive biomarkers in ALS, with particular emphasis on those that are the most promising ones for clinical trial design and routine clinical practice. Neurofilaments in cerebrospinal fluid and blood are the main prognostic and pharmacodynamic biomarkers. Furthermore, several candidates exist covering various pathological aspects of the disease, such as immune, metabolic and muscle damage markers. Urine has been studied less often and should be explored for its possible advantages. New advances in the knowledge of cryptic exons introduce the possibility of discovering new biomarkers. Collaborative efforts, prospective studies and standardized procedures are needed to validate candidate biomarkers. A combined biomarkers panel can provide a more detailed disease status.

Funder

Instituto de Salud Carlos III

European Regional Development Fund

Publisher

MDPI AG

Subject

General Medicine

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