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

• 批准号: 9129578
• 负责人: ROBERT P BOWSER
• 金额: $ 0.5万
• 依托单位: IRON HORSE DIAGNOSTICS, INC.
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-06-01 至 2018-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9129578
• 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; Health; 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; diagnostic assay; experience; nervous system disorder; neuromuscular; prospective; research study

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）：肌萎缩性侧面硬化症（ALS或Lou Gehrig病）是一种致命的神经退行性疾病，典型的寿命为诊断后3-5年。最初的症状可能类似于许多其他神经系统疾病，通常从肢体弱点，肌肉束缚或言语或呼吸困难开始。目前尚无针对ALS的确定诊断测试，临床诊断通常是 症状发作需要12个月的时间，并依靠排除其他临床症状的其他潜在原因。较早的诊断将允许使用较新的药物更快地开始治疗 发育中的疗法或同样重要的疗法提供了一种方法，可以快速排除ALS作为症状的原因。创建了Iron Horse Diagnostics，Inc。，目的是最终确定ALS的诊断和预后指标的开发，并将这些测定法推销为实验室开发的测试（LDT）在临床实验室改进修正案（CLIA）认证实验室中。我们已经生产了一系列经过同行评审的出版物，令人信服地证明，在ALS患者的血液和CSF中可以检测到显着水平的PNFH和补体C3，并且我们的测定水平为ALS提供了诊断效用。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测定法，这是商业化之前的最后剩余步骤。 这是一个FastTrack应用程序，因为阶段III之前的其余步骤用高度可定义的GO/No-Go门进行了很好的定义。在拟议工作的第一阶段，我们将通过测试指示性蛋白质并将测定技术转换为新平台，将测定作为ALS的诊断指标作为ALS的诊断指标。第二阶段将通过进行多中心的前瞻性临床研究研究，并在CLIA认证的实验室中使用患者样品进行测定，以确定ALS诊断的整体准确性。该提案的目的是为这些生物标志物生成敏感且经过验证的LDT（实验室开发的测试），可用于CLIA认证实验室中的临床诊断ALS。经过认证的LDT将以服务为临床医生。