Bowser Robert, Lacomis David
Department of Pathology, University of Pittsburgh School of Medicine, BST S-420, 200 Lothrop Street, Pittsburgh, Pennsylvania 15261, USA.
Muscle Nerve. 2009 Nov;40(5):753-62. doi: 10.1002/mus.21488.
Protein-based biomarkers for amyotrophic lateral sclerosis (ALS) and other motor neuron diseases (MNDs) have many potential clinical utilities, including diagnostic, prognostic, and drug development indications. During the past decade a number of potential protein biomarkers have been proposed for MNDs. Further verification studies, followed by large validation and qualification studies, are required to advance these initial discoveries toward clinical use. Study of additional patient populations, including disease mimics, is required during the validation phase of biomarker development. Important regulatory issues are discussed that will affect the timing and strategy for biomarker assay development in ALS and other MNDs. The continued development of protein biomarkers for MNDs requires extensive collaboration between academic clinicians and scientists in conjunction with the biotechnology and pharmaceutical industries.
用于肌萎缩侧索硬化症（ALS）和其他运动神经元疾病（MNDs）的基于蛋白质的生物标志物具有许多潜在的临床用途，包括诊断、预后和药物开发指征。在过去十年中，已经提出了一些用于MNDs的潜在蛋白质生物标志物。需要进一步的验证研究，随后进行大规模的验证和鉴定研究，以便将这些初步发现推进到临床应用阶段。在生物标志物开发的验证阶段，需要对包括疾病模拟物在内的更多患者群体进行研究。讨论了将影响ALS和其他MNDs生物标志物检测方法开发的时间和策略的重要监管问题。MNDs蛋白质生物标志物的持续开发需要学术临床医生和科学家与生物技术和制药行业广泛合作。
