ALS biomarkers for therapy development

State of the field and future directions

Michael G Benatar, Kevin Boylan, Andreas Jeromin, Seward B. Rutkove, James Berry, Nazem Atassi, Lucie Bruijn

Research output: Contribution to journalArticle

28 Citations (Scopus)

Abstract

Biomarkers have become the focus of intense research in the field of amyotrophic lateral sclerosis (ALS), with the hope that they might aid therapy development efforts. Notwithstanding the discovery of many candidate biomarkers, none have yet emerged as validated tools for drug development. In this review we present a nuanced view of biomarkers based on the perspective of the Food and Drug Administration; highlight the distinction between discovery and validation; describe existing and emerging resources; review leading biological fluid-based, electrophysiological, and neuroimaging candidates relevant to therapy development efforts; discuss lessons learned from biomarker initiatives in related neurodegenerative diseases; and outline specific steps that we, as a field, might take to hasten the development and validation of biomarkers that will prove useful in enhancing efforts to develop effective treatments for ALS patients. Most important among these is the proposal to establish a federated ALS Biomarker Consortium in which all interested and willing stakeholders may participate with equal opportunity to contribute to the broader mission of biomarker development and validation.

Original languageEnglish (US)
Pages (from-to)169-182
Number of pages14
JournalMuscle and Nerve
Volume53
Issue number2
DOIs
StatePublished - Feb 1 2016

Fingerprint

Amyotrophic Lateral Sclerosis
Biomarkers
Therapeutics
United States Food and Drug Administration
Direction compound
Neuroimaging
Neurodegenerative Diseases
Research
Pharmaceutical Preparations

Keywords

  • ALS
  • Biomarkers
  • Disease progression
  • Pharmacodynamic
  • Predictive
  • Prognostic

ASJC Scopus subject areas

  • Clinical Neurology
  • Cellular and Molecular Neuroscience
  • Physiology (medical)
  • Physiology

Cite this

Benatar, M. G., Boylan, K., Jeromin, A., Rutkove, S. B., Berry, J., Atassi, N., & Bruijn, L. (2016). ALS biomarkers for therapy development: State of the field and future directions. Muscle and Nerve, 53(2), 169-182. https://doi.org/10.1002/mus.24979

ALS biomarkers for therapy development : State of the field and future directions. / Benatar, Michael G; Boylan, Kevin; Jeromin, Andreas; Rutkove, Seward B.; Berry, James; Atassi, Nazem; Bruijn, Lucie.

In: Muscle and Nerve, Vol. 53, No. 2, 01.02.2016, p. 169-182.

Research output: Contribution to journalArticle

Benatar, MG, Boylan, K, Jeromin, A, Rutkove, SB, Berry, J, Atassi, N & Bruijn, L 2016, 'ALS biomarkers for therapy development: State of the field and future directions', Muscle and Nerve, vol. 53, no. 2, pp. 169-182. https://doi.org/10.1002/mus.24979
Benatar, Michael G ; Boylan, Kevin ; Jeromin, Andreas ; Rutkove, Seward B. ; Berry, James ; Atassi, Nazem ; Bruijn, Lucie. / ALS biomarkers for therapy development : State of the field and future directions. In: Muscle and Nerve. 2016 ; Vol. 53, No. 2. pp. 169-182.
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