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Utility of Dissociated Intrinsic Hand Muscle Atrophy in the Diagnosis of Amyotrophic Lateral Sclerosis
Published on: March 4, 2014
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Longitudinal biomarkers in amyotrophic lateral sclerosis
Fen Huang1, Yuda Zhu1, Jennifer Hsiao-Nakamoto1
1Denali Therapeutics, South San Francisco, California, USA.
Annals of Clinical and Translational Neurology
|June 10, 2020
Summary
Biomarkers like cytokines and neurofilaments are elevated in amyotrophic lateral sclerosis (ALS) and can predict disease progression. These markers are stable over time, aiding treatment monitoring.
Area of Science:
- Neuroscience
- Immunology
- Biomarker Discovery
Background:
- Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease.
- Identifying reliable biomarkers for ALS progression and treatment response is crucial.
Purpose of the Study:
- Investigate neurodegenerative and inflammatory biomarkers in people with ALS (PALS).
- Evaluate their predictive value for ALS progression rates.
- Assess their utility as pharmacodynamic biomarkers for monitoring treatment effects.
Main Methods:
- Analyzed longitudinal plasma and cerebrospinal fluid (CSF) samples from PALS and controls.
- Stratified PALS into fast- and slow-progression subgroups.
- Compared cytokine and neurofilament (NF) levels between groups and in relation to C9orf72 mutations.
Main Results:
- Elevated cytokines (MCP-1, IL-18) and neurofilaments (NFs) found in PALS compared to controls.
- Higher levels of these biomarkers in fast-progressing PALS and those with C9orf72 mutations.
- CSF/plasma neurofilament light chain (NFL) levels predict disease progression and can stratify patient groups.
Conclusions:
- Cytokines and NFs are elevated in PALS, indicating neurodegeneration and inflammation.
- Longitudinal stability of these biomarkers supports their use in monitoring treatment effects.
- NFs have prognostic value and can aid in selecting patient subsets for clinical trials.

