Commercialization of a Diagnostic test for amyotrophic lateral sclerosis (ALS)

肌萎缩侧索硬化症 (ALS) 诊断测试的商业化

基本信息

批准号：
8735259
负责人：
ROBERT P BOWSER
金额：
$ 81.36万
依托单位：
IRON HORSE DIAGNOSTICS, INC.
依托单位国家：
美国
项目类别：
财政年份：
2013
资助国家：
美国
起止时间：
2013-09-01 至 2015-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8735259
关键词：
Age Amendment Amyotrophic Lateral Sclerosis Antibodies Biological Assay Biological Markers Blood Blood specimen Breathing Cerebrospinal Fluid Clinic Clinical Clinical Research Complement Development Diagnosis Diagnostic Diagnostic tests Disease Early Diagnosis Enzyme-Linked Immunosorbent Assay Equipment Equus caballus Evaluation Goals Immunoassay Iron Laboratories Limb structure Longevity Marketing Measures Motor Muscle fasciculation Muscular Atrophy Neurodegenerative Disorders Neurologist Neuropathy Paralysed Patient Care Patients Peer Review Pharmacotherapy Phase Production Prognostic Marker Proteins Publications Qualifying Reagent Sampling Sensitivity and Specificity Series Services Speech Symptoms Technology Testing Time Translating Work base clinical Diagnosis commercialization diagnostic accuracy experience nervous system disorder neuromuscular prospective public health relevance research study

项目摘要

DESCRIPTION (provided by applicant): Amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) is a fatal neurodegenerative disease with a typical lifespan of 3 - 5 years after diagnosis. The initial symptoms can be similar to many other neurologic disorders and often begin with limb weakness, muscle fasciculations, or increasing difficulties in speech or breathing. No definitive diagnostic tests currently exist for ALS, and clinical diagnosis typically takes 12 months from symptom onset and relies on ruling out other potential causes of the clinical symptoms. Earlier diagnosis will permit more rapid initiation of treatment with newer drug therapies in development, or equally important provide a means to rapidly rule out ALS as a cause of the symptoms. Iron Horse Diagnostics, Inc. was created to finalize the development of diagnostic and prognostic indicators of ALS and market these assays as laboratory developed tests (LDTs) in a Clinical Laboratory Improvement Amendments (CLIA) certified lab. We have produced a series of peer-reviewed publications convincingly demonstrating that significant levels of pNfH and complement c3 can be detected in the blood and CSF of ALS patients, and that the levels detected by our assays provide diagnostic utility for ALS. We propose a series of tasks to optimize and validate our assays in a CLIA certified laboratory, the last remaining steps before commercialization. This is a FastTrack application because the remaining steps before Phase III are well defined with highly definable go/no-go gates. During Phase I of the proposed work, we will finalize and optimize assays as diagnostic indicators of ALS by testing samples for indicative proteins and translating the assay technology onto a new platform. Phase II will expand on this by conducting a multi-center prospective clinical research study and qualifying the assays using patient samples in a CLIA-certified lab to determine the overall accuracy of the ALS diagnostic. The goal of this proposal is to generate a sensitive and validated LDT (Lab Developed Test) for these biomarkers useful for clinically diagnosing ALS in a CLIA certified lab. A certified LDT will be marketed to clinicians as a service.

描述（由申请人提供）：肌萎缩侧索硬化症（ALS 或 Lou Gehrig 病）是一种致命的神经退行性疾病，诊断后典型寿命为 3 - 5 年。最初的症状可能与许多其他神经系统疾病相似，通常始于四肢无力、肌肉颤动或言语或呼吸困难。目前尚无针对 ALS 的明确诊断测试，通常需要临床诊断从症状出现起需要 12 个月，并且依赖于排除临床症状的其他潜在原因。早期诊断将允许更快地开始新药治疗正在开发的疗法，或者同样重要的是，提供了一种快速排除 ALS 症状原因的方法。 Iron Horse Diagnostics, Inc. 的成立是为了完成 ALS 诊断和预后指标的开发，并在临床实验室改进修正案 (CLIA) 认证的实验室中将这些检测方法作为实验室开发的测试 (LDT) 进行销售。我们发表了一系列经过同行评审的出版物，令人信服地证明，可以在 ALS 患者的血液和脑脊液中检测到显着水平的 pNfH 和补体 c3，并且我们的检测检测到的水平为 ALS 提供了诊断实用性。我们提出了一系列任务，以在 CLIA 认证的实验室中优化和验证我们的检测，这是商业化之前的最后剩余步骤。这是一个 FastTrack 应用程序，因为第三阶段之前的剩余步骤已通过高度可定义的通过/不通过门进行了明确定义。在拟议工作的第一阶段，我们将通过测试样本中的指示性蛋白质并将检测技术转化为新平台来最终确定和优化作为 ALS 诊断指标的检测方法。 II 期将在此基础上进行扩展，开展多中心前瞻性临床研究，并在 CLIA 认证的实验室中使用患者样本对检测进行鉴定，以确定 ALS 诊断的整体准确性。该提案的目标是为这些生物标志物生成灵敏且经过验证的 LDT（实验室开发测试），可用于在 CLIA 认证实验室中临床诊断 ALS。经过认证的 LDT 将作为一项服务向临床医生销售。