Fluid-Based Biomarkers for Amyotrophic Lateral Sclerosis.

Area of Science:

• Neuroscience
• Biochemistry
• Clinical Research

Background:

• Amyotrophic lateral sclerosis (ALS) is a complex neurodegenerative disease lacking effective treatments.
• Drug development for ALS is hindered by the absence of reliable biomarkers for diagnosis, progression monitoring, and treatment efficacy assessment.
• Fluid-based biomarkers offer a promising avenue to address these unmet needs in ALS research.

Purpose of the Study:

• To review the current state of biomarker discovery for amyotrophic lateral sclerosis (ALS).
• To highlight biomarkers advancing towards clinical utility in ALS diagnosis and treatment.
• To discuss the challenges in validating and implementing ALS biomarkers in clinical practice.

Main Methods:

• Review of recent advancements in cerebrospinal fluid, serum, plasma, urine, and saliva biomarkers for ALS.
• Analysis of biomarker applications in patient stratification, disease course prediction, and preclinical/clinical studies.
• Assessment of the progress and hurdles in translating ALS biomarker research into clinical practice.

Main Results:

• Significant advancements have been made in identifying potential fluid-based biomarkers for ALS.
• Several biomarker candidates show promise for patient stratification and predicting disease progression.
• Despite progress, the validation and clinical implementation of ALS biomarkers remain challenging.

Conclusions:

• Fluid-based biomarkers hold significant potential for improving the diagnosis, monitoring, and treatment of amyotrophic lateral sclerosis (ALS).
• Further research and rigorous validation are essential to overcome current challenges and translate promising biomarker candidates into clinical tools for ALS drug development.
• Addressing biomarker challenges is critical for accelerating the development of effective therapies for ALS.