Alzheimer's prevention initiative: a proposal to evaluate presymptomatic treatments as quickly as possible

Biomark Med. 2010 Feb;4(1):3-14. doi: 10.2217/bmm.09.91.


Now is the time to launch the era of Alzheimer's disease (AD) prevention research, establish the methods and infrastructure to rapidly evaluate presymptomatic AD treatments and evaluate them rigorously and rapidly in randomized clinical trials. This article is a call to arms. It contends that the evaluation of presymptomatic AD treatments must become an urgent priority, it identifies what is holding us back and proposes new public policies and scientific strategies to overcome these roadblocks. It defines the term 'presymptomatic AD treatment', notes the best established biomarkers of AD progression and pathology and suggests how they could be used to rapidly evaluate presymptomatic AD treatments in the people at risk. It introduces an approach to evaluate presymptomatic AD treatments in asymptomatic people at the highest risk of imminent clinical onset and determines the extent to which the treatment's biomarker predicts a clinical outcome. We propose an Alzheimer's Prevention Initiative, which is now being reviewed and refined in partnership with leading academic and industry investigators. It is intended to evaluate the most promising presymptomatic AD treatments, help develop a regulatory pathway for their accelerated approval using reasonably likely surrogate end points and find demonstrably effective presymptomatic AD treatments as quickly as possible.

Keywords: MRI; PET; amyloid; apolipoprotein; biomarker; clinical trial; genetics; presenilin; public policy; surrogate marker.

Publication types

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

MeSH terms

  • Alzheimer Disease / diagnosis
  • Alzheimer Disease / drug therapy
  • Alzheimer Disease / prevention & control*
  • Amyloid beta-Peptides / cerebrospinal fluid
  • Apolipoprotein E4 / genetics
  • Biomarkers / cerebrospinal fluid
  • Humans
  • Magnetic Resonance Imaging
  • Nootropic Agents / therapeutic use
  • Positron-Emission Tomography
  • Public Policy
  • Randomized Controlled Trials as Topic
  • United States
  • tau Proteins / cerebrospinal fluid


  • Amyloid beta-Peptides
  • Apolipoprotein E4
  • Biomarkers
  • MAPT protein, human
  • Nootropic Agents
  • tau Proteins