A major goal of the neuroscience community is to develop treatment strategies that will slow or forestall the progression of chronic neurodegenerative diseases. Parkinson's disease (PD) is one of the most common adult neurodegenerative disorders, affecting over 1 million people in North America and the European Union, As a first step in identifying such therapies, the NINDS Exploratory Trials in Parkinson's disease (NET-PD) network successfully completed futility studies, which identified creatine as a potential agent to slow clinical decline in PD. The NET-PD network is now conducting a large, long-term, Phase 3 trial (known as LSI) comparing creatine to placebo. An additional futility study is underway (the FS-ZONE study) to examine the potential for pioglitazone as a disease modifying therapy in PD.

Public Health Relevance

The accumulated disability that PD causes is a major source of diminished quality of life and increased health care costs. Despite the advances in our understanding of the pathophysiology in PD, there are no current therapies that slow the inexorable clinical decline. LSI will help to determine if creatine can slow clinical decline and provide better information on the course of early PD than is currently available.

Agency
National Institute of Health (NIH)
Institute
National Institute of Neurological Disorders and Stroke (NINDS)
Type
Cooperative Clinical Research--Cooperative Agreements (U10)
Project #
2U10NS044482-11
Application #
8460593
Study Section
Special Emphasis Panel (ZNS1-SRB-B (34))
Program Officer
Moy, Claudia S
Project Start
2002-09-30
Project End
2015-11-30
Budget Start
2013-01-01
Budget End
2013-11-30
Support Year
11
Fiscal Year
2013
Total Cost
$76,285
Indirect Cost
$51,806
Name
Beth Israel Deaconess Medical Center
Department
Type
DUNS #
071723621
City
Boston
State
MA
Country
United States
Zip Code
02215
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Xiao, J; Zhao, Y; Bastian, R W et al. (2010) Novel THAP1 sequence variants in primary dystonia. Neurology 74:229-38