Change in MRI striatal volumes as a biomarker in preclinical Huntington's disease

Research output: Contribution to journalArticle

Abstract

This article discusses the need for biomarkers and surrogate endpoints for future clinical trials in individuals at risk for Huntington's disease. Definitions and criteria are presented for biomarkers and surrogate endpoints, and data are presented suggesting that striatal volumes, as measured on MRI scans, meet the criteria for a biomarker. Biomarkers can be used in lieu of clinical endpoints in treatment trials if there is evidence that treatment affects the biomarker in a way that is predictive of endpoint status. Because there are currently no effective treatments for Huntington's disease, it is not yet possible to validate whether change in MRI striatal volumes can serve as an effective surrogate endpoint. It is recommended that future clinical trials be designed using MRI striatal volumes to "screen" potential treatments. Those treatments that reduce the rate of striatal atrophy can then be tested with delay of symptom onset as the clinical endpoint. This strategy is essential if efficient and cost-effective clinical trials are to be conducted in the preclinical stage of Huntington's disease.

Original languageEnglish (US)
Pages (from-to)152-158
Number of pages7
JournalBrain Research Bulletin
Volume72
Issue number2-3 SPEC. ISS.
DOIs
StatePublished - Apr 30 2007
Externally publishedYes

Fingerprint

Corpus Striatum
Huntington Disease
Biomarkers
Clinical Trials
Atrophy
Magnetic Resonance Imaging
Costs and Cost Analysis

Keywords

  • Biomarker
  • Clinical trial
  • Huntington's disease
  • Preclinical
  • Surrogate endpoint

ASJC Scopus subject areas

  • Neuroscience(all)

Cite this

Change in MRI striatal volumes as a biomarker in preclinical Huntington's disease. / Aylward, Elizabeth H.

In: Brain Research Bulletin, Vol. 72, No. 2-3 SPEC. ISS., 30.04.2007, p. 152-158.

Research output: Contribution to journalArticle

@article{bb73fe6397584b63a1f8420f7d845918,
title = "Change in MRI striatal volumes as a biomarker in preclinical Huntington's disease",
abstract = "This article discusses the need for biomarkers and surrogate endpoints for future clinical trials in individuals at risk for Huntington's disease. Definitions and criteria are presented for biomarkers and surrogate endpoints, and data are presented suggesting that striatal volumes, as measured on MRI scans, meet the criteria for a biomarker. Biomarkers can be used in lieu of clinical endpoints in treatment trials if there is evidence that treatment affects the biomarker in a way that is predictive of endpoint status. Because there are currently no effective treatments for Huntington's disease, it is not yet possible to validate whether change in MRI striatal volumes can serve as an effective surrogate endpoint. It is recommended that future clinical trials be designed using MRI striatal volumes to {"}screen{"} potential treatments. Those treatments that reduce the rate of striatal atrophy can then be tested with delay of symptom onset as the clinical endpoint. This strategy is essential if efficient and cost-effective clinical trials are to be conducted in the preclinical stage of Huntington's disease.",
keywords = "Biomarker, Clinical trial, Huntington's disease, Preclinical, Surrogate endpoint",
author = "Aylward, {Elizabeth H.}",
year = "2007",
month = "4",
day = "30",
doi = "10.1016/j.brainresbull.2006.10.028",
language = "English (US)",
volume = "72",
pages = "152--158",
journal = "Brain Research Bulletin",
issn = "0361-9230",
publisher = "Elsevier Inc.",
number = "2-3 SPEC. ISS.",

}

TY - JOUR

T1 - Change in MRI striatal volumes as a biomarker in preclinical Huntington's disease

AU - Aylward, Elizabeth H.

PY - 2007/4/30

Y1 - 2007/4/30

N2 - This article discusses the need for biomarkers and surrogate endpoints for future clinical trials in individuals at risk for Huntington's disease. Definitions and criteria are presented for biomarkers and surrogate endpoints, and data are presented suggesting that striatal volumes, as measured on MRI scans, meet the criteria for a biomarker. Biomarkers can be used in lieu of clinical endpoints in treatment trials if there is evidence that treatment affects the biomarker in a way that is predictive of endpoint status. Because there are currently no effective treatments for Huntington's disease, it is not yet possible to validate whether change in MRI striatal volumes can serve as an effective surrogate endpoint. It is recommended that future clinical trials be designed using MRI striatal volumes to "screen" potential treatments. Those treatments that reduce the rate of striatal atrophy can then be tested with delay of symptom onset as the clinical endpoint. This strategy is essential if efficient and cost-effective clinical trials are to be conducted in the preclinical stage of Huntington's disease.

AB - This article discusses the need for biomarkers and surrogate endpoints for future clinical trials in individuals at risk for Huntington's disease. Definitions and criteria are presented for biomarkers and surrogate endpoints, and data are presented suggesting that striatal volumes, as measured on MRI scans, meet the criteria for a biomarker. Biomarkers can be used in lieu of clinical endpoints in treatment trials if there is evidence that treatment affects the biomarker in a way that is predictive of endpoint status. Because there are currently no effective treatments for Huntington's disease, it is not yet possible to validate whether change in MRI striatal volumes can serve as an effective surrogate endpoint. It is recommended that future clinical trials be designed using MRI striatal volumes to "screen" potential treatments. Those treatments that reduce the rate of striatal atrophy can then be tested with delay of symptom onset as the clinical endpoint. This strategy is essential if efficient and cost-effective clinical trials are to be conducted in the preclinical stage of Huntington's disease.

KW - Biomarker

KW - Clinical trial

KW - Huntington's disease

KW - Preclinical

KW - Surrogate endpoint

UR - http://www.scopus.com/inward/record.url?scp=33847607138&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=33847607138&partnerID=8YFLogxK

U2 - 10.1016/j.brainresbull.2006.10.028

DO - 10.1016/j.brainresbull.2006.10.028

M3 - Article

C2 - 17352939

AN - SCOPUS:33847607138

VL - 72

SP - 152

EP - 158

JO - Brain Research Bulletin

JF - Brain Research Bulletin

SN - 0361-9230

IS - 2-3 SPEC. ISS.

ER -