Sampling, biomarker OPtimization and Harmonization In ALS (SOPHIA)

ALS 中的采样、生物标志物优化和协调 (SOPHIA)

• 批准号: MR/K000780/1
• 负责人: Martin Turner
• 金额: $ 9.31万
• 依托单位: University of Oxford
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2012
• 资助国家: 英国
• 起止时间: 2012 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=MR%2FK000780%2F1
• 关键词: Sampling; biomarker; OPtimization; Harmonization; ALS

Amyotrophic Lateral Sclerosis (ALS) is one of the most devastating diseases in neurology affecting some 50,000 individuals at any time in Europe, and causing around 10,000 deaths each year. The main clinical features are weakness and wasting of muscles, but dementia may also occur. ALS represents a good model for study of all neurodegenerative conditions, as it has a characteristic phenotype, rapid progression and the correlation between diagnosis during life and autopsy diagnosis is close to 100%. However, validated neurochemical biomarkers for monitoring disease activity, for generating earlier diagnoses and for defining prognosis are lacking. Active European collaborations are in place for harmonizing clinical datasets, neuroimaging and neuropathology protocols. A preliminary strategy for harmonization of biological and tissue samples has been established. Standardized protocols for clinical data and sample collection are now urgently required for optimization and harmonization of biomarker development. The overall aim of this proposal is to provide a common European strategy for the prioritization and selection of candidate biomarker domains for optimization and harmonization. This will in turn provide a long-term platform by which existing collaborative structures that are relevant to neurodegenerative disease biomarkers (including academic initiatives, co-funding strategies, biobanks, industrial efforts, private-public alliances) are integrated within an inclusive web-based virtual biobank. Samples and clinical/imaging/neurophysiologic and neuropathological datasets provided by participating members can then be optimally utilized to enable state of the art collaborative analyses.The established platform will also act as an important communication channel between this consortium and the rest of the ALS/Neurodegeneration field to ensure that the optimization efforts are in line with the whole ALS/ND field, to avoid duplication of work, and to ensure better acceptance and utilization of the project results by all stakeholders. Ultimately, the platform will be used to disseminate the results to the whole ALS/Neurodegeneration field, and will act as a permanent Interactive European ALS biomarker platform for researchers to optimize/harmonize novel biomarkers using an established pan-European ALS methodology. The platform will also allow interaction with those of other cognate groups (e.g the NEALS group within the US) and with patient groups and other relevant stakeholders.

肌萎缩侧索硬化症 (ALS) 是神经病学中最具破坏性的疾病之一，在欧洲任何时候都会影响约 50,000 人，每年造成约 10,000 人死亡。主要临床特征是肌肉无力和萎缩，但也可能发生痴呆。 ALS 代表了研究所有神经退行性疾病的良好模型，因为它具有特征性表型、快速进展，并且生前诊断与尸检诊断之间的相关性接近 100%。然而，缺乏用于监测疾病活动、进行早期诊断和确定预后的经过验证的神经化学生物标志物。欧洲正在积极开展合作，以协调临床数据集、神经影像学和神经病理学协议。已经制定了生物和组织样品协调的初步策略。现在迫切需要临床数据和样本收集的标准化方案来优化和协调生物标志物的开发。该提案的总体目标是为候选生物标志物域的优先级排序和选择提供共同的欧洲策略，以实现优化和协调。这反过来将提供一个长期平台，通过该平台将与神经退行性疾病生物标志物相关的现有合作结构（包括学术倡议、共同资助战略、生物库、工业努力、公私联盟）整合到一个包容性的基于网络的虚拟生物库中。参与成员提供的样本以及临床/影像/神经生理学和神经病理学数据集可以得到最佳利用，以实现最先进的协作分析。所建立的平台还将充当该联盟与 ALS/神经退行性疾病领域其他成员之间的重要沟通渠道，以确保优化工作与整个 ALS/ND 领域保持一致，避免重复工作，并确保更好的接受和认可 所有利益相关者对项目成果的利用。最终，该平台将用于将结果传播到整个 ALS/神经退行性疾病领域，并将作为永久的交互式欧洲 ALS 生物标志物平台，供研究人员使用已建立的泛欧洲 ALS 方法来优化/协调新型生物标志物。该平台还将允许与其他同源团体（例如美国的 NEALS 团体）以及患者团体和其他相关利益相关者进行互动。

项目成果

Diagnostic accuracy of diffusion tensor imaging in amyotrophic lateral sclerosis: a systematic review and individual patient data meta-analysis.

• DOI: 10.1016/j.acra.2013.03.017
• 发表时间: 2013-09
• 期刊: ACADEMIC RADIOLOGY
• 影响因子: 4.8
• 作者: Foerster, Bradley R.;Dwamena, Ben A.;Petrou, Myria;Carlos, Ruth C.;Callaghan, Brian C.;Churchill, Cristina L.;Mohamed, Mona A.;Bartels, Claudia;Benatar, Michael;Bonzano, Laura;Ciccarelli, Olga;Cosottini, Mirco;Ellis, Cathy M.;Ehrenreich, Hannelore;Filippini, Nicola;Ito, Mizuki;Kalra, Sanjay;Melhem, Elias R.;Pyra, Timothy;Roccatagliata, Luca;Senda, Joe;Sobue, Gen;Turner, Martin R.;Feldman, Eva L.;Pomper, Martin G.
• 通讯作者: Pomper, Martin G.

The ALSFRS as an outcome measure in therapeutic trials and its relationship to symptom onset.

• DOI: 10.3109/21678421.2016.1140786
• 发表时间: 2016-07
• 期刊: Amyotrophic lateral sclerosis & frontotemporal degeneration
• 影响因子: 2.8
• 作者: Proudfoot M;Jones A;Talbot K;Al-Chalabi A;Turner MR
• 通讯作者: Turner MR

Progressive hemiparesis (Mills syndrome) with aphasia in amyotrophic lateral sclerosis.

• DOI: 10.1212/wnl.0000000000000090
• 发表时间: 2014-02-04
• 期刊: Neurology
• 影响因子: 9.9
• 作者: Bäumer D;Butterworth R;Menke RA;Talbot K;Hofer M;Turner MR
• 通讯作者: Turner MR

Imaging as a biomarker in drug discovery for Alzheimer's disease: is MRI a suitable technology?

• DOI: 10.1186/alzrt276
• 发表时间: 2014
• 期刊: Alzheimer's research & therapy
• 作者: Merlo Pich E;Jeromin A;Frisoni GB;Hill D;Lockhart A;Schmidt ME;Turner MR;Mondello S;Potter WZ
• 通讯作者: Potter WZ

Neurofilament light chain: A prognostic biomarker in amyotrophic lateral sclerosis.

• DOI: 10.1212/wnl.0000000000001642
• 发表时间: 2015-06-02
• 期刊: Neurology
• 影响因子: 9.9
• 作者: Lu CH;Macdonald-Wallis C;Gray E;Pearce N;Petzold A;Norgren N;Giovannoni G;Fratta P;Sidle K;Fish M;Orrell R;Howard R;Talbot K;Greensmith L;Kuhle J;Turner MR;Malaspina A
• 通讯作者: Malaspina A