Parkinson's Disease Neuroprotection Clinical Center

描述（由申请人提供）：帕金森病（PD）是一种影响大约一百万美国人的神经退行性疾病。所有患者均出现逐渐进行性的体征和症状组合，包括震颤、强直、行动迟缓、流口水、低音量说话、缩微症、步态和平衡困难以及日常生活活动困难。众所周知，PD患者具有从黑质向纹状体投射的含有多巴胺的神经元的丢失，大多数临床体征和症状对多巴胺能药物替代治疗有反应。左旋多巴，多巴胺激动剂)。然而，神经退行性变的潜在机制尚不清楚，因此，迄今为止，没有治疗方法可以治愈或减缓疾病的进展。基于体外研究和动物数据，许多治疗方法被假设为具有神经保护作用，但临床试验尚未发现这些药物对PD患者具有神经保护作用。缺乏神经保护治疗对患者、护理人员、卫生专业人员和经济来说都是一个严重的问题。随着疾病的进展，护理费用增加，估计每年的医疗费用从疾病初期每年3 000美元增加到疾病晚期每年15 000美元。因此，寻找一种能够减缓或阻止帕金森病进展的药物是帕金森病研究的主要目标。为此，NIH设立了一个多中心研究小组，对潜在的神经保护剂进行临床试验。该应用程序的具体目标是成为NIH神经保护治疗试验临床中心之一。该申请建议在梅奥诊所和太阳健康研究所建立一个中心联盟，以便能够最好地招募早期PD患者进行神经保护治疗试验。拟议的联盟计划在42个月内每月招募2名患者，预计将达到这些招募目标。梅奥诊所斯科茨代尔，梅奥诊所杰克逊维尔，梅奥诊所罗切斯特和太阳健康研究所，都有专门护理和治疗PD患者的临床中心，都有良好的临床试验经验。这些资源可用于在这四个地点进行临床试验，由10名专门治疗帕金森病的神经科医生组成的专职医生，以及所需的护理人员和物理设备。这些方案将由各个临床中心的pi与NIH的工作人员一起制定。

DESCRIPTION (provided by applicant): Parkinson's disease (PD) is a neurodegenerative disorder affecting approximately one million Americans. All patients develop a gradually progressive combination of signs and symptoms, including tremor, rigidity, slowness of movement, drooling, low volume speech, micrographia, gait and balance difficulty, and difficulty with activities of daily living. It is known that patients with PD have a loss of dopamine containing neurons projecting from the substantia nigra to the striatum, and most clinical signs and symptoms respond to replacement therapy with doparminergic medications (ie. levodopa, dopamine agonists). However, the underlying mechanism of neurodegeneration is unknown and therefore, to date, there is no treatment to cure or slow the progression of the disease. Many treatments have been hypothesized to be neuroprotective based on in vitro studies and animal data, but clinical trials have not found these drugs to be neuroprotective in patients with PD. The lack of a neuroprotective treatment has been a serious problem for patients, caregivers, health professionals, and for the economy. As the disease progresses the cost of care increases, with estimates of annual cost for medical care going from $3,000/yr early in the disease course to $15,000/yr in later stage disease. Finding a medication(s) that will slow or halt the progression of PD is therefore a major goal of PD research. To this end creating a multi-center study group to run clinical trials of potential neuroprotective agents has been set as a goal of the NIH. This application has, as its specific aim, to be one of the NIH Clinical Centers for neuroprotection treatment trials. This application proposes to create a consortium of centers at the Mayo Clinic and Sun Health Research Institute in order to best be able to enroll patients with early PD for neuroprotection treatment trials. With the mandate to enroll two patients/month for 42 months, the proposed consortium expects to meet these recruitment goals. Mayo Clinic Scottsdale, Mayo Clinic Jacksonville, Mayo Clinic Rochester, and Sun Health Research Institute, all have clinical centers that specialize in the care and treatment of patients with PD, and all have well established clinical trials experience. The resources are available to run clinical trials with a dedicated physician staff of 10 neurologists specializing in treatment of Parkinson's Disease at these four sites, along with nursing staff and the physical plant required. The protocols will be developed by the PIs of the various clinical centers along with NIH staff.