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Biofluid Biomarkers in Huntington's Disease
Filipe B Rodrigues1, Lauren M Byrne1, Edward J Wild2
1Huntington's Disease Centre, Department of Neurodegenerative Disease, Institute of Neurology, University College London, London, UK.
Insights
Huntington's disease (HD) research needs better biomarkers for tracking disease progression and symptoms. Biofluid biomarkers offer a promising avenue for reliable, molecular-level insights to advance clinical trials and drug development for HD.
Area of Science:
- Neurodegenerative diseases
- Biomarker discovery
- Molecular diagnostics
Background:
- Huntington's disease (HD) is a progressive neurodegenerative disorder lacking effective disease-modifying treatments.
- Current limitations in managing HD stem from a lack of reliable indicators for disease severity, progression, and phenotype.
- There is a critical need for new markers to guide clinical trials and therapeutic development.
Purpose of the Study:
- To review existing literature on biofluid biomarkers for Huntington's disease.
- To identify biofluid biomarkers with the highest potential for clinical applicability in HD management.
- To highlight the advantages of biofluid biomarkers in assessing disease progression and facilitating drug development.
Main Methods:
- Comprehensive review of published reports on biofluid biomarker studies in Huntington's disease.
- Emphasis on biomarkers demonstrating reliability, reproducibility, accuracy, and direct molecular quantification.
- Assessment of biomarker potential for clinical trial empowerment and hypothesis generation.
Main Results:
- Biofluid biomarkers offer advantages over traditional clinical measures, including cost-effectiveness and precision.
- These biomarkers can directly quantify underlying pathobiological processes at a molecular level.
- Several biofluid markers show promise for clinical application in tracking HD progression.
Conclusions:
- Biofluid biomarkers are crucial for advancing Huntington's disease research and clinical practice.
- The identified biomarkers are key to improving the reliability of clinical trials and developing new therapeutic strategies.
- Further development and validation of these biofluid markers are essential for effective HD patient management.
Abstract:
Huntington's disease (HD) is a chronic progressive neurodegenerative condition where new markers of disease progression are needed. So far no disease-modifying interventions have been found, and few interventions have been proven to alleviate symptoms. This may be partially explained by the lack of reliable indicators of disease severity, progression, and phenotype.Biofluid biomarkers may bring advantages in addition to clinical measures, such as reliability, reproducibility, price, accuracy, and direct quantification of pathobiological processes at the molecular level; and in addition to empowering clinical trials, they have the potential to generate useful hypotheses for new drug development.In this chapter we review biofluid biomarker reports in HD, emphasizing those we feel are likely to be closest to clinical applicability.