Cerebrospinal fluid biomarkers for Alzheimer's disease: A view of the regulatory science qualification landscape from the coalition against major diseases CSF biomarker team

Stephen P. Arnerić, Richard Batrla-Utermann, Laurel A Beckett, Tobias Bittner, Kaj Blennow, Leslie Carter, Robert Dean, Sebastiaan Engelborghs, Just Genius, Mark Forrest Gordon, Janice Hitchcock, June Kaplow, Johan Luthman, Richard Meibach, David Raunig, Klaus Romero, Mahesh N. Samtani, Mary Savage, Leslie Shaw, Diane StephensonRobert M. Umek, Hugo Vanderstichele, Brian Willis, Susan Yule

Research output: Contribution to journalReview article

20 Scopus citations

Abstract

Alzheimer's disease (AD) drug development is burdened with the current requirement to conduct large, lengthy, and costly trials to overcome uncertainty in patient progression and effect size on treatment outcome measures. There is an urgent need for the discovery, development, and implementation of novel, objectively measured biomarkers for AD that would aid selection of the appropriate subpopulation of patients in clinical trials, and presumably, improve the likelihood of successfully evaluating innovative treatment options. Amyloid deposition and tau in the brain, which are most commonly assessed either in cerebrospinal fluid (CSF) or by molecular imaging, are consistently and widely accepted. Nonetheless, a clear gap still exists in the accurate identification of subjects that truly have the hallmarks of AD. The Coalition Against Major Diseases (CAMD), one of 12 consortia of the Critical Path Institute (C-Path), aims to streamline drug development for AD and related dementias by advancing regulatory approved drug development tools for clinical trials through precompetitive data sharing and adoption of consensus clinical data standards. This report focuses on the regulatory process for biomarker qualification, briefly comments on how it contrasts with approval or clearance of companion diagnostics, details the qualifications currently available to the field of AD, and highlights the current challenges facing the landscape of CSF biomarkers qualified as hallmarks of AD. Finally, it recommends actions to accelerate regulatory qualification of CSF biomarkers that would, in turn, improve the efficiency of AD therapeutic development.

Original languageEnglish (US)
Pages (from-to)19-35
Number of pages17
JournalJournal of Alzheimer's Disease
Volume55
Issue number1
DOIs
StatePublished - 2016

Keywords

  • Alzheimer's disease
  • biomarker qualification
  • cerebrospinal fluid biomarkers
  • Coalition Against Major Diseases

ASJC Scopus subject areas

  • Clinical Psychology
  • Geriatrics and Gerontology
  • Psychiatry and Mental health

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    Arnerić, S. P., Batrla-Utermann, R., Beckett, L. A., Bittner, T., Blennow, K., Carter, L., Dean, R., Engelborghs, S., Genius, J., Gordon, M. F., Hitchcock, J., Kaplow, J., Luthman, J., Meibach, R., Raunig, D., Romero, K., Samtani, M. N., Savage, M., Shaw, L., ... Yule, S. (2016). Cerebrospinal fluid biomarkers for Alzheimer's disease: A view of the regulatory science qualification landscape from the coalition against major diseases CSF biomarker team. Journal of Alzheimer's Disease, 55(1), 19-35. https://doi.org/10.3233/JAD-160573