Parkinson's Disease Neuroprotection Clinical Center

描述（由申请人提供）：帕金森病（PD）是一种神经退行性疾病，影响约一百万美国人。所有患者均出现逐渐进展的体征和症状组合，包括震颤、僵硬、运动缓慢、流口水、低音量言语、显微书写、步态和平衡困难以及日常生活活动困难。众所周知，PD患者从黑质投射到纹状体的含多巴胺神经元丢失，大多数临床体征和症状对多巴酚丁胺能药物（即多巴胺能药物）的替代治疗有反应。左旋多巴、多巴胺激动剂）。然而，神经变性的潜在机制是未知的，因此，迄今为止，没有治疗方法来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早期的3,000美元/年增加到疾病晚期的15,000美元/年。因此，找到一种减缓或阻止PD进展的药物是PD研究的主要目标。为此，建立一个多中心研究小组来进行潜在的神经保护剂的临床试验已经成为NIH的目标。作为其具体目标，该申请是NIH神经保护治疗试验的临床中心之一。本申请建议在马约诊所和Sun Health Research Institute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。根据42个月的每月招募2名患者的要求，拟议的联盟预计将达到这些招募目标。马约诊所斯科茨代尔、马约诊所杰克逊维尔、马约诊所罗切斯特和Sun Health Research Institute都有专门从事PD患者护理和治疗的临床中心，并且都有完善的临床试验经验。这些资源可用于在这四个地点与10名专门治疗帕金森病的神经科医生组成的专职医生工作人员进行临床试验，沿着还有所需的护理人员和物理设备。方案将由各临床中心的PI与NIH工作人员沿着制定。

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.