Rosenthal Liana S, Drake Daniel, Alcalay Roy N, Babcock Debra, Bowman F DuBois, Chen-Plotkin Alice, Dawson Ted M, Dewey Richard B, German Dwight C, Huang Xuemei, Landin Barry, McAuliffe Matthew, Petyuk Vladislav A, Scherzer Clemens R, Hillaire-Clarke Coryse St, Sieber Beth-Anne, Sutherland Margaret, Tarn Chi, West Andrew, Vaillancourt David, Zhang Jing, Gwinn Katrina
Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
Department of Biostatistics, Columbia University, New York, New York, USA.
Mov Disord. 2016 Jun;31(6):915-23. doi: 10.1002/mds.26438. Epub 2015 Oct 7.
Neuroprotection for Parkinson's disease (PD) remains elusive. Biomarkers hold the promise of removing roadblocks to therapy development. The National Institute of Neurological Disorders and Stroke has therefore established the Parkinson's Disease Biomarkers Program to promote discovery of PD biomarkers for use in phase II and III clinical trials.
Using a novel consortium design, the Parkinson's Disease Biomarker Program is focused on the development of clinical and laboratory-based biomarkers for PD diagnosis, progression, and prognosis. Standardized operating procedures and pooled reference samples were created to allow cross-project comparisons and assessment of batch effects. A web-based Data Management Resource facilitates rapid sharing of data and biosamples across the research community for additional biomarker projects.
Eleven consortium projects are ongoing, seven of which recruit participants and obtain biosamples. As of October 2014, 1,082 participants have enrolled (620 PD, 101 with other causes of parkinsonism, 23 essential tremor, and 338 controls), 1,040 of whom have at least one biosample. Six thousand eight hundred ninety-eight total biosamples are available from baseline, 6-, 12-, and 18-month visits: 1,006 DNA, 1,661 RNA, 1,419 whole blood, 1,382 plasma, 1,200 serum, and 230 cerebrospinal fluid (CSF). Quality control analysis of plasma, serum, and CSF samples indicates that almost all samples are high quality (24 of 2,812 samples exceed acceptable hemoglobin levels).
By making samples and data widely available, using stringent operating procedures based on existing standards, hypothesis testing for biomarker discovery, and providing a resource that complements existing programs, the Parkinson's Disease Biomarker Program will accelerate the pace of PD biomarker research. © 2015 International Parkinson and Movement Disorder Society.
帕金森病（PD）的神经保护治疗仍然难以实现。生物标志物有望消除治疗开发中的障碍。因此，美国国立神经疾病和中风研究所设立了帕金森病生物标志物计划，以促进用于II期和III期临床试验的PD生物标志物的发现。
帕金森病生物标志物计划采用新颖的联盟设计，专注于开发基于临床和实验室的生物标志物，用于PD的诊断、病情进展和预后评估。制定了标准化操作程序和汇总参考样本，以允许跨项目比较和批次效应评估。基于网络的数据管理资源有助于在研究社区内快速共享数据和生物样本，以支持其他生物标志物项目。
11个联盟项目正在进行中，其中7个项目招募参与者并获取生物样本。截至2014年10月，已有1082名参与者入组（620例PD患者、101例患有其他帕金森综合征病因者、23例特发性震颤患者和338例对照），其中1040人至少有一份生物样本。从基线、6个月、12个月和18个月随访中总共获得了6898份生物样本：1006份DNA、1661份RNA、1419份全血、1382份血浆、1200份血清和230份脑脊液（CSF）。血浆、血清和CSF样本的质量控制分析表明，几乎所有样本质量都很高（2812份样本中有24份超过可接受的血红蛋白水平）。
通过广泛提供样本和数据、采用基于现有标准的严格操作程序、进行生物标志物发现的假设检验以及提供补充现有项目的资源，帕金森病生物标志物计划将加快PD生物标志物研究的步伐。©2015国际帕金森和运动障碍协会。
