I-Corps: Early Stage Diagnosis of Parkinson's Disease

I-Corps：帕金森病的早期诊断

• 批准号: 1911946
• 负责人: Zhihao Zhuang
• 金额: $ 5万
• 依托单位: University of Delaware
• 依托单位国家: 美国
• 项目类别: Standard Grant
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-02-15 至 2020-01-31
• 项目状态: 已结题
• 来源: https://www.nsf.gov/awardsearch/showAward?AWD_ID=1911946&HistoricalAwards=false
• 关键词: Corps; Early; Stage; Diagnosis; Parkinson

The broader impact/commercial potential of this I-Corps project is to provide a biological measure of Parkinsons disease (PD) which is extremely valuable in determining treatment strategies for PD patients and in drug development against PD. PD is the second most common neurodegenerative disease in the United States with treatment costs that exceeded $14.4 billion in 2010 and projected to double by 2040. Effective treatment of PD requires reliable early diagnosis of PD, however no objective test for definitive diagnosis of PD currently exists. Current diagnosis is based on outward symptoms like tremors, slow movement, rigid muscle, brain imaging and usually requires multiple visits with neurology specialists to avoid misdiagnosis. In fact, a 2013 study determined that the direct cost of diagnosis for a new PD patient on average amounts to $7,322 and with 60,000 new diagnosis of PD made every year in US, the market of PD diagnosis is estimated to be a minimum $0.4 billion. A new diagnostic approach for early stage of Parkinson's disease (PD) will have a tremendous impact on the prevention and treatment of PD by providing a stratification strategy of patients and increase the success in clinical trial of PD treatment. This I-Corps project is based on an understanding of the role of protein homeostasis in the cell and developing tools and techniques to decipher these pathways. PD is a neurological disease with the key hallmark of large deposits of Lewy bodies, an amalgam of aggregated proteins, in the affected motor neurons. Literature reports have identified certain biomarkers in Lewy bodies that have unique signature of post-translational modification which can cross the blood brain barrier. This project leverages techniques that identify signatures on the biomarker in PD blood samples and utilize tools that recombinantly generate biomarkers and develop antibodies against them, which will allow development of a test kit for early detection of PD non-invasively.This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.

这个i-Corps项目的更广泛的影响/商业潜力是提供帕金森氏病(PD)的生物学测量，这在确定PD患者的治疗策略和治疗PD的药物开发方面非常有价值。帕金森病是美国第二常见的神经退行性疾病，2010年的治疗费用超过144亿美元，预计到2040年将翻一番。帕金森病的有效治疗需要可靠的帕金森病早期诊断，但目前尚无明确诊断帕金森病的客观测试。目前的诊断基于震颤、运动缓慢、肌肉僵硬、脑成像等外在症状，通常需要多次去看神经科专家，以避免误诊。事实上，2013年的一项研究确定，一个新的帕金森氏症患者的直接诊断成本平均为7,322美元，而美国每年新诊断的帕金森氏症患者有60,000人，帕金森氏症诊断市场估计至少为4亿美元。一种新的帕金森病早期诊断方法将对帕金森病的预防和治疗产生巨大的影响，它将提供患者的分层策略，并增加帕金森病治疗的临床试验的成功率。这个i-Corps项目是基于对细胞中蛋白质稳态的作用的理解，以及开发工具和技术来破译这些途径。帕金森病是一种神经系统疾病，其主要特征是在受影响的运动神经元中大量沉积路易小体，这是一种聚集蛋白质的混合物。文献报道在路易小体中发现了某些生物标志物，它们具有独特的翻译后修饰特征，可以穿越血脑屏障。该项目利用识别帕金森病血液样本中生物标记物上的签名的技术，并利用重组生成生物标记物并开发针对它们的抗体的工具，这将允许开发一种检测试剂盒，用于非侵入性地早期检测帕金森病。该奖项反映了NSF的法定使命，并通过使用基金会的智力优势和更广泛的影响审查标准进行评估，被认为值得支持。