Protein Biomarkers for Amyotrophic Lateral Sclerosis

Authors and Disclosures

Henrik Ryberg, PhD, University of Pittsburgh School of Medicine, Department of Pathology, Center for ALS Research; Pittsburgh Institute for Neurodegenerative Diseases, Pittsburgh, PA 15261, USA Tel.: +1 412 648 9655 Fax: +1 412 648 1916 her15@pitt.edu
Robert Bowser, PhD, University of Pittsburgh School of Medicine, Department of Pathology, Center for ALS Research; Pittsburgh Institute for Neurodegenerative Diseases, Pittsburgh, PA 15261, USA Tel.: +1 412 383 7819 Fax: +1 412 648 1916 bowserrp@upmc.edu

Financial & competing interests disclosure: The corresponding author (Robert Bowser) is a cofounder of Knopp Neurosciences Inc., a biotechnology company focused on the development of therapeutics for ALS and other neurodegenerative diseases. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
No writing assistance was utilized in the production of this manuscript.

Sidebar: Key Issues

• Standardized acquisition, processing and storage methods for biofluids are required to obtain reliable proteomics data during the discovery phase.

• Owing to the heterogeneous nature of amyotrophic lateral sclerosis (ALS), panels of protein biomarkers derived from cerebrospinal fluid and blood may be most valuable for generating an accurate diagnostic test for ALS. Protein-based biomarkers may help determine the overall heterogeneity within the disease.

• Additional biofluids, such as urine and saliva, should also be pursued with respect to biomarker discovery efforts.

• Longitudinal studies are required to identify surrogate biomarkers for ALS disease progression. Such biomarkers may identify protein alterations that correlate to slow or more rapid disease progression.

• Most biomarker discovery efforts have utilized too few subjects in the study design. Increased collaboration between academic and industry investigators is required to generate larger experimental sample sizes in order to both discover and validate ALS-specific biomarkers.