Protein biomarker discovery and validation: the long and uncertain path to clinical utility

Nat Biotechnol. 2006 Aug;24(8):971-83. doi: 10.1038/nbt1235.

Abstract

Better biomarkers are urgently needed to improve diagnosis, guide molecularly targeted therapy and monitor activity and therapeutic response across a wide spectrum of disease. Proteomics methods based on mass spectrometry hold special promise for the discovery of novel biomarkers that might form the foundation for new clinical blood tests, but to date their contribution to the diagnostic armamentarium has been disappointing. This is due in part to the lack of a coherent pipeline connecting marker discovery with well-established methods for validation. Advances in methods and technology now enable construction of a comprehensive biomarker pipeline from six essential process components: candidate discovery, qualification, verification, research assay optimization, biomarker validation and commercialization. Better understanding of the overall process of biomarker discovery and validation and of the challenges and strategies inherent in each phase should improve experimental study design, in turn increasing the efficiency of biomarker development and facilitating the delivery and deployment of novel clinical tests.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Biomarkers / blood*
  • Blood Chemical Analysis / methods*
  • Blood Proteins / analysis*
  • Humans
  • Mass Spectrometry / methods*
  • Peptide Mapping / methods*
  • Protein Array Analysis / methods*
  • Sensitivity and Specificity

Substances

  • Biomarkers
  • Blood Proteins