Parkinson's Disease Neuroprotection Clinical Center

描述(申请人提供)：帕金森氏病(PD)是一种神经退行性疾病，影响大约100万美国人。所有患者都逐渐出现症状和体征的组合，包括震颤、僵硬、行动迟缓、流口水、低语量、微字症、步态和平衡困难，以及日常生活活动困难。众所周知，帕金森病患者有从黑质向纹状体投射的含有多巴胺的神经元的丢失，大多数临床症状和体征对多巴胺能药物的替代治疗有反应(即。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，到目前为止，还没有治愈或减缓疾病进展的治疗方法。根据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帕金森氏症患者具有神经保护作用。缺乏神经保护性治疗一直是患者、护理人员、卫生专业人员和经济的一个严重问题。随着疾病的发展，护理费用增加，估计每年的医疗费用从病程早期的3,000美元/年到晚期疾病的15,000美元/年。因此，找到一种药物(S)可以减缓或阻止帕金森病的进展是帕金森病研究的主要目标。为此，创建一个多中心研究小组对潜在的神经保护剂进行临床试验已被定为美国国立卫生研究院的目标。这项申请的具体目标是成为美国国立卫生研究院神经保护治疗试验临床中心之一。这项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，以便最好地招募早期帕金森病患者参加神经保护治疗试验。由于规定每月招收两名患者，为期42个月，拟议的财团预计将实现这些招募目标。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帕金森氏症患者的临床中心，并且都有良好的临床试验经验。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团队以及所需的护理人员和物理设备进行临床试验。这些方案将由各个临床中心的PI与NIH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.