Neurotherapeutics

, Volume 14, Issue 1, pp 119–134 | Cite as

Fluid-Based Biomarkers for Amyotrophic Lateral Sclerosis

Review

Abstract

Amyotrophic lateral sclerosis (ALS) is a highly heterogeneous disease with no effective treatment. Drug development has been hampered by the lack of biomarkers that aid in early diagnosis, demonstrate target engagement, monitor disease progression, and can serve as surrogate endpoints to assess the efficacy of treatments. Fluid-based biomarkers may potentially address these issues. An ideal biomarker should exhibit high specificity and sensitivity for distinguishing ALS from control (appropriate disease mimics and other neurologic diseases) populations and monitor disease progression within individual patients. Significant progress has been made using cerebrospinal fluid, serum, and plasma in the search for ALS biomarkers, with urine and saliva biomarkers still in earlier stages of development. A few of these biomarker candidates have demonstrated use in patient stratification, predicting disease course (fast vs slow progression) and severity, or have been used in preclinical and clinical applications. However, while ALS biomarker discovery has seen tremendous advancements in the last decade, validating biomarkers and moving them towards the clinic remains more elusive. In this review, we highlight biomarkers that are moving towards clinical utility and the challenges that remain in order to implement biomarkers at all stages of the ALS drug development process.

Keywords

ALS Biomarkers Prognostic Diagnostic Clinical Preclinical 

Supplementary material

13311_2016_503_MOESM1_ESM.pdf (533 kb)
ESM 1(PDF 533 kb)

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Copyright information

© The American Society for Experimental NeuroTherapeutics, Inc. 2016

Authors and Affiliations

  1. 1.Department of NeurologyBarrow Neurological Institute, St. Joseph’s Hospital and Medical CenterPhoenixUSA
  2. 2.Department of NeurobiologyBarrow Neurological Institute, St. Joseph’s Hospital and Medical CenterPhoenixUSA

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