Biofluid Biomarkers in the Prognosis of Amyotrophic Lateral Sclerosis : Recent Developments and Therapeutic Applications
Sánchez-Tejerina, Daniel (Hospital Universitari Vall d'Hebron)
Llaurado, Arnau (Hospital Universitari Vall d'Hebron)
Sotoca Fernández, Javier (Hospital Universitari Vall d'Hebron)
Lopez-Diego, Veronica (Hospital Universitari Vall d'Hebron)
Vidal Taboada, Jose M. (Hospital Universitari Vall d'Hebron)
Salvado, Maria (Hospital Universitari Vall d'Hebron)
Juntas Morales, Raúl (Hospital Universitari Vall d'Hebron)
Universitat Autònoma de Barcelona

Data:	2023
Resum:	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.
Drets:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.
Llengua:	Anglès
Document:	Article ; recerca ; Versió publicada
Matèria:	Amyotrophic lateral sclerosis (ALS) ; Biomarker ; Genetics ; Neuroinflammation ; Neurofilament light (NfL) protein ; Pharmacodynamic biomarker ; Prognosis
Publicat a:	Cells, Vol. 12 (april 2023) , ISSN 2073-4409

DOI: 10.3390/cells12081180
PMID: 37190090

19 p, 365.1 KB

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