Prevention trials in Alzheimer's disease: An EU-US task force report

Bruno Vellas, Paul S. Aisen, Cristina Sampaio, Maria Carrillo, Philip Scheltens, Bruno Scherrer, Giovanni B. Frisoni, Michael Weiner, Lon Schneider, Serge Gauthier, Christine C Gispen de Wied, Suzanne Hendrix, Howard Feldman, Jesse Cedarbaum, Ronald Carl Petersen, Eric Siemers, Sandrine Andrieu, David Prvulovic, Jacques Touchon, Harald Hampel

Research output: Contribution to journalArticle

44 Citations (Scopus)

Abstract

Despite enormous financial and scientific efforts, still no approved disease-modifying therapies exist for Alzheimer's disease (AD). During the last decade all Phase III clinical trials on disease modifiers in AD have failed. The dementia stage of AD being probably too late in order to allow for successful disease modification has been identified as a possible culprit that could explain the failure of so many clinical trials. In parallel, a major development in the diagnostic research field of AD was achieved by the recent proposal of new diagnostic criteria for AD, which also specifically incorporate the use of biomarkers as defining criteria for preclinical stages of AD, thus extending the traditional definition of disease to very early stages that may be a more feasible target for various disease modifying therapeutic interventions. This ongoing paradigm shift in AD definition and diagnosis represents a fundamental basis for redefinition of interventional trials in AD, allowing to specifically focus on preventative measures during very early pathophysiologically confirmed stages of disease. This consensus paper reflects the outcome from a European Union and North American Task Force meeting comprised of experts from academia, industry, private foundations, and regulatory agencies that was convened in Toulouse, France on November 5, 2010 and that focused on prevention trials in AD. This position paper thoroughly analyzes prerequisites for successful preventative trials in AD and concludes with concrete recommendations on biomarkers, statistical tools and other variables important for improved study designs suitable for preventative as well as for early therapeutic interventional trials in AD.

Original languageEnglish (US)
Pages (from-to)594-600
Number of pages7
JournalProgress in Neurobiology
Volume95
Issue number4
DOIs
StatePublished - Dec 2011

Fingerprint

Advisory Committees
Alzheimer Disease
Biomarkers
Phase III Clinical Trials
European Union
France
Dementia
Consensus
Industry
Therapeutics
Clinical Trials

Keywords

  • Alzheimer's disease
  • Biomarkers
  • Early intervention trials
  • Prevention trials
  • Study design
  • Study planning

ASJC Scopus subject areas

  • Neuroscience(all)

Cite this

Vellas, B., Aisen, P. S., Sampaio, C., Carrillo, M., Scheltens, P., Scherrer, B., ... Hampel, H. (2011). Prevention trials in Alzheimer's disease: An EU-US task force report. Progress in Neurobiology, 95(4), 594-600. https://doi.org/10.1016/j.pneurobio.2011.08.014

Prevention trials in Alzheimer's disease : An EU-US task force report. / Vellas, Bruno; Aisen, Paul S.; Sampaio, Cristina; Carrillo, Maria; Scheltens, Philip; Scherrer, Bruno; Frisoni, Giovanni B.; Weiner, Michael; Schneider, Lon; Gauthier, Serge; Wied, Christine C Gispen de; Hendrix, Suzanne; Feldman, Howard; Cedarbaum, Jesse; Petersen, Ronald Carl; Siemers, Eric; Andrieu, Sandrine; Prvulovic, David; Touchon, Jacques; Hampel, Harald.

In: Progress in Neurobiology, Vol. 95, No. 4, 12.2011, p. 594-600.

Research output: Contribution to journalArticle

Vellas, B, Aisen, PS, Sampaio, C, Carrillo, M, Scheltens, P, Scherrer, B, Frisoni, GB, Weiner, M, Schneider, L, Gauthier, S, Wied, CCGD, Hendrix, S, Feldman, H, Cedarbaum, J, Petersen, RC, Siemers, E, Andrieu, S, Prvulovic, D, Touchon, J & Hampel, H 2011, 'Prevention trials in Alzheimer's disease: An EU-US task force report', Progress in Neurobiology, vol. 95, no. 4, pp. 594-600. https://doi.org/10.1016/j.pneurobio.2011.08.014
Vellas B, Aisen PS, Sampaio C, Carrillo M, Scheltens P, Scherrer B et al. Prevention trials in Alzheimer's disease: An EU-US task force report. Progress in Neurobiology. 2011 Dec;95(4):594-600. https://doi.org/10.1016/j.pneurobio.2011.08.014
Vellas, Bruno ; Aisen, Paul S. ; Sampaio, Cristina ; Carrillo, Maria ; Scheltens, Philip ; Scherrer, Bruno ; Frisoni, Giovanni B. ; Weiner, Michael ; Schneider, Lon ; Gauthier, Serge ; Wied, Christine C Gispen de ; Hendrix, Suzanne ; Feldman, Howard ; Cedarbaum, Jesse ; Petersen, Ronald Carl ; Siemers, Eric ; Andrieu, Sandrine ; Prvulovic, David ; Touchon, Jacques ; Hampel, Harald. / Prevention trials in Alzheimer's disease : An EU-US task force report. In: Progress in Neurobiology. 2011 ; Vol. 95, No. 4. pp. 594-600.
@article{35700fc2a58a4079b42e89db1024d355,
title = "Prevention trials in Alzheimer's disease: An EU-US task force report",
abstract = "Despite enormous financial and scientific efforts, still no approved disease-modifying therapies exist for Alzheimer's disease (AD). During the last decade all Phase III clinical trials on disease modifiers in AD have failed. The dementia stage of AD being probably too late in order to allow for successful disease modification has been identified as a possible culprit that could explain the failure of so many clinical trials. In parallel, a major development in the diagnostic research field of AD was achieved by the recent proposal of new diagnostic criteria for AD, which also specifically incorporate the use of biomarkers as defining criteria for preclinical stages of AD, thus extending the traditional definition of disease to very early stages that may be a more feasible target for various disease modifying therapeutic interventions. This ongoing paradigm shift in AD definition and diagnosis represents a fundamental basis for redefinition of interventional trials in AD, allowing to specifically focus on preventative measures during very early pathophysiologically confirmed stages of disease. This consensus paper reflects the outcome from a European Union and North American Task Force meeting comprised of experts from academia, industry, private foundations, and regulatory agencies that was convened in Toulouse, France on November 5, 2010 and that focused on prevention trials in AD. This position paper thoroughly analyzes prerequisites for successful preventative trials in AD and concludes with concrete recommendations on biomarkers, statistical tools and other variables important for improved study designs suitable for preventative as well as for early therapeutic interventional trials in AD.",
keywords = "Alzheimer's disease, Biomarkers, Early intervention trials, Prevention trials, Study design, Study planning",
author = "Bruno Vellas and Aisen, {Paul S.} and Cristina Sampaio and Maria Carrillo and Philip Scheltens and Bruno Scherrer and Frisoni, {Giovanni B.} and Michael Weiner and Lon Schneider and Serge Gauthier and Wied, {Christine C Gispen de} and Suzanne Hendrix and Howard Feldman and Jesse Cedarbaum and Petersen, {Ronald Carl} and Eric Siemers and Sandrine Andrieu and David Prvulovic and Jacques Touchon and Harald Hampel",
year = "2011",
month = "12",
doi = "10.1016/j.pneurobio.2011.08.014",
language = "English (US)",
volume = "95",
pages = "594--600",
journal = "Progress in Neurobiology",
issn = "0301-0082",
publisher = "Elsevier Limited",
number = "4",

}

TY - JOUR

T1 - Prevention trials in Alzheimer's disease

T2 - An EU-US task force report

AU - Vellas, Bruno

AU - Aisen, Paul S.

AU - Sampaio, Cristina

AU - Carrillo, Maria

AU - Scheltens, Philip

AU - Scherrer, Bruno

AU - Frisoni, Giovanni B.

AU - Weiner, Michael

AU - Schneider, Lon

AU - Gauthier, Serge

AU - Wied, Christine C Gispen de

AU - Hendrix, Suzanne

AU - Feldman, Howard

AU - Cedarbaum, Jesse

AU - Petersen, Ronald Carl

AU - Siemers, Eric

AU - Andrieu, Sandrine

AU - Prvulovic, David

AU - Touchon, Jacques

AU - Hampel, Harald

PY - 2011/12

Y1 - 2011/12

N2 - Despite enormous financial and scientific efforts, still no approved disease-modifying therapies exist for Alzheimer's disease (AD). During the last decade all Phase III clinical trials on disease modifiers in AD have failed. The dementia stage of AD being probably too late in order to allow for successful disease modification has been identified as a possible culprit that could explain the failure of so many clinical trials. In parallel, a major development in the diagnostic research field of AD was achieved by the recent proposal of new diagnostic criteria for AD, which also specifically incorporate the use of biomarkers as defining criteria for preclinical stages of AD, thus extending the traditional definition of disease to very early stages that may be a more feasible target for various disease modifying therapeutic interventions. This ongoing paradigm shift in AD definition and diagnosis represents a fundamental basis for redefinition of interventional trials in AD, allowing to specifically focus on preventative measures during very early pathophysiologically confirmed stages of disease. This consensus paper reflects the outcome from a European Union and North American Task Force meeting comprised of experts from academia, industry, private foundations, and regulatory agencies that was convened in Toulouse, France on November 5, 2010 and that focused on prevention trials in AD. This position paper thoroughly analyzes prerequisites for successful preventative trials in AD and concludes with concrete recommendations on biomarkers, statistical tools and other variables important for improved study designs suitable for preventative as well as for early therapeutic interventional trials in AD.

AB - Despite enormous financial and scientific efforts, still no approved disease-modifying therapies exist for Alzheimer's disease (AD). During the last decade all Phase III clinical trials on disease modifiers in AD have failed. The dementia stage of AD being probably too late in order to allow for successful disease modification has been identified as a possible culprit that could explain the failure of so many clinical trials. In parallel, a major development in the diagnostic research field of AD was achieved by the recent proposal of new diagnostic criteria for AD, which also specifically incorporate the use of biomarkers as defining criteria for preclinical stages of AD, thus extending the traditional definition of disease to very early stages that may be a more feasible target for various disease modifying therapeutic interventions. This ongoing paradigm shift in AD definition and diagnosis represents a fundamental basis for redefinition of interventional trials in AD, allowing to specifically focus on preventative measures during very early pathophysiologically confirmed stages of disease. This consensus paper reflects the outcome from a European Union and North American Task Force meeting comprised of experts from academia, industry, private foundations, and regulatory agencies that was convened in Toulouse, France on November 5, 2010 and that focused on prevention trials in AD. This position paper thoroughly analyzes prerequisites for successful preventative trials in AD and concludes with concrete recommendations on biomarkers, statistical tools and other variables important for improved study designs suitable for preventative as well as for early therapeutic interventional trials in AD.

KW - Alzheimer's disease

KW - Biomarkers

KW - Early intervention trials

KW - Prevention trials

KW - Study design

KW - Study planning

UR - http://www.scopus.com/inward/record.url?scp=82755189703&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=82755189703&partnerID=8YFLogxK

U2 - 10.1016/j.pneurobio.2011.08.014

DO - 10.1016/j.pneurobio.2011.08.014

M3 - Article

C2 - 21925234

AN - SCOPUS:82755189703

VL - 95

SP - 594

EP - 600

JO - Progress in Neurobiology

JF - Progress in Neurobiology

SN - 0301-0082

IS - 4

ER -