A MULTICENTER STUDY FOR THE VALIDATION OF ALS BIOMARKERS

验证 ALS 生物标志物的多中心研究

基本信息

批准号：
8166934
负责人：
TAHSEEN MOZAFFAR
金额：
$ 0.03万
依托单位：
UNIVERSITY OF CALIFORNIA, SAN DIEGO
依托单位国家：
美国
项目类别：
财政年份：
2009
资助国家：
美国
起止时间：
2009-12-01 至 2010-06-30
项目状态：
已结题

项目摘要

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The primary objective is to identify factors that contribute to the pathogenesis of amyotrophic lateral sclerosis (ALS). Development of disease biomarkers and diagnostic laboratory tests would facilitate earlier treatment intervention, help monitor treatment efficacy; and, ultimately, lead to the identification of targets that could be used in therapy development. Specific aim 1 is to develop a laboratory test that can be used by primary care physicians, neurologists and other clinicians to provide guidance with regard to something to worry about (possible ALS). It would provide benefit in generating appropriate referrals to neurological specialists early in disease progression. False negatives could be tolerated but false positives cannot (i.e. high specificity required with acceptable sensitivity). The test will identify people with ALS and distinguish ALS from disorders that present with similar symptoms (e.g. carpal tunnel, radiculopathies, myopathies). A test with high sensitivity for ALS can be very useful for specialty neurologist as well for early and accurate detection for inclusion in clinical trials. Blood and CSF samples will be collected at multiple sites representing the four regions of the United States from age and gender matched healthy individuals, disease mimics, and ALS patients. Specific aim 2 is to assess how biomarkers discovered in aim 1 change with disease course. Blood samples will be collected longitudinally from ALS and suspected ALS volunteers.

该子项目是利用该技术的众多研究子项目之一资源由 NIH/NCRR 资助的中心拨款提供。子项目及研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金，因此可以在其他 CRISP 条目中表示。列出的机构是对于中心来说，它不一定是研究者的机构。主要目标是确定导致肌萎缩侧索硬化症 (ALS) 发病机制的因素。疾病生物标志物和诊断实验室测试的开发将有助于早期治疗干预，有助于监测治疗效果；并最终确定可用于治疗开发的靶标。具体目标 1 是开发一种可供初级保健医生、神经科医生和其他临床医生使用的实验室测试，以就需要担心的事情（可能的 ALS）提供指导。它将有助于在疾病进展的早期向神经学专家进行适当的转诊。可以容忍假阴性，但不能容忍假阳性（即需要高特异性和可接受的灵敏度）。该测试将识别 ALS 患者，并将 ALS 与具有类似症状的疾病（例如腕管、神经根病、肌病）区分开来。高灵敏度 ALS 测试对于专业神经科医生以及早期准确检测以纳入临床试验非常有用。血液和脑脊液样本将在代表美国四个地区的多个地点从年龄和性别匹配的健康个体、疾病模拟者和 ALS 患者中采集。具体目标 2 是评估目标 1 中发现的生物标志物如何随病程变化。将从 ALS 和疑似 ALS 志愿者中纵向采集血液样本。