Why has therapy development for dementia failed in the last two decades?

Serge Gauthier, Marilyn Albert, Nick Fox, Michel Goedert, Miia Kivipelto, Jorge Mestre-Ferrandiz, Lefkos T. Middleton

Research output: Contribution to journalShort surveypeer-review

84 Scopus citations


The success rate of the pharmaceutical research and development (R&D) for dementia drugs has been abysmally low, in the last two decades. Also low has been the number of pipeline drugs in development, compared to other therapy areas. However, the rationale of early terminations has not been reported in the majority of trials. These are key findings of the recently published pharmaceutical pipeline analysis by the UK-based Office of Health Economics (OHE). Our understanding of main challenges include (1) the significant gaps of knowledge in the nosology and complexity of the underpinning biological mechanisms of the commonest, not familial, forms of late onset dementias; (2) low signal-to-noise ratio, notwithstanding the lack of validated biomarkers as entry and/or end-point criteria; (3) recruitment and retention, particularly in the asymptomatic and early disease stages. A number of current and future strategies aimed at ameliorating drug development are outlined and discussed.

Original languageEnglish (US)
Pages (from-to)60-64
Number of pages5
JournalAlzheimer's and Dementia
Issue number1
StatePublished - Jan 1 2016


  • Alzheimer's
  • Amyloid hypothesis
  • Attrition
  • Clinical trials
  • Dementia
  • Research & development

ASJC Scopus subject areas

  • Clinical Neurology
  • Geriatrics and Gerontology
  • Psychiatry and Mental health
  • Cellular and Molecular Neuroscience
  • Health Policy
  • Developmental Neuroscience
  • Epidemiology


Dive into the research topics of 'Why has therapy development for dementia failed in the last two decades?'. Together they form a unique fingerprint.

Cite this