Biofluid Core

摘要 – ARTFL LEFFTDS 纵向 FTLD：生物流体核心 额颞叶变性 (FTLD) 的成功治疗需要早期准确的诊断， 以及在临床试验中监测新干预措施效果的能力。 ALLFTD 的总体目标是 生成具有良好特征的潜在临床试验参与者群体，他们拥有与临床相关的数据 试验纳入和分层标准，来自血液或脑脊液的液体生物标志物数据对此很有用。而且， 随着越来越多的 FTLD 临床试验即将进行，迫切需要改进现有液体 生物标志物并开发具有更高灵敏度和特异性的新工具，以阐明下游影响 干预措施。除了生成流体生物标志物数据外，生物流体核心的一个主要作用是管理 生物样本和生物标志物数据共享以及帮助管理协作生物标志物发现工作 与学术界和工业界。生物流体核心将由 FTLD 生物标记物专家领导。伦纳德 Petrucelli 和 Adam Boxer 博士是脑脊液神经退行性生物标志物发现和分析方面的专家。 Anne Fagan，实现以下目标：（1）监督生物体液标本的采集、处理、储存和 与临床核心、行政核心和国家集中存储库合作共享 阿尔茨海默病和相关痴呆症 (NCRAD) 生物库。 (2) 执行标准测量 所有参与者脑脊液和血液中神经退行性变的生物标志物，以及有针对性的测量 特定 f-FTLD 群体中的遗传相关蛋白。 （3）支持外部合作，识别新的 FTLD 治疗靶点和生物标志物。

ABSTRACT – ARTFL LEFFTDS Longitudinal FTLD: BIOFLUID CORE Successful treatment of Frontotemporal Lobar Degeneration (FTLD) will require early and accurate diagnosis, and the ability to monitor the effects of novel interventions in clinical trials. An overarching goal of ALLFTD is to generate a well-characterized population of potential clinical trial participants who have data relevant to clinical trial inclusion and stratification criteria, for which fluid biomarker data from blood or CSF are useful. Moreover, as increasing numbers of clinical trials for FTLD are imminent, there is an urgent need to refine current fluid biomarkers and develop new tools with improved sensitivity and specificity to elucidate downstream effects of interventions. In addition to generating fluid biomarker data, a major role of the Biofluid Core will be to manage biospecimen and biomarker data sharing as well as to help manage collaborative biomarker discovery efforts with academia and industry. The Biofluid Core will be led by experts in FTLD biomarkers, Drs. Leonard Petrucelli and Adam Boxer, and an expert in CSF neurodegenerative biomarker discovery and analysis, Dr. Anne Fagan, to achieve the following aims: (1) Oversee biofluid specimen collection, processing, storage and sharing in collaboration with the Clinical Core, Administrative Core and the National Centralized Repository for Alzheimer's Disease and Related Dementias (NCRAD) biobank. (2) Perform measurements of standard biomarkers of neurodegeneration in CSF and blood in all participants, and targeted measurements of genetically relevant proteins in specific f-FTLD populations. (3) Support external collaborations to identify new FTLD therapeutic targets and biomarkers.