Gulf South IPF Clinical Research Network

描述(由申请人提供)： 特发性肺纤维化(IPF)是一种使人衰弱和致命的疾病，目前还没有已知的治疗方法。最近的流行病学研究发现，IPF比以前认识到的更常见，据估计，美国每年有30,000例新诊断病例。最近有许多专家评论提醒我们注意以前推荐的治疗方案的有效性缺乏证据，这些方案仅依靠皮质类固醇与其他免疫抑制剂或不结合其他免疫抑制剂来治疗IPF。现在是时候测试来自NIH、ALA和私人基金会数十年来对临床和基础科学肺纤维化研究的支持的新方法的有效性了。这项建议的总体目标是检验这一假设，即预防疾病进展的最有效的治疗将需要多种药物，并基于以下知识：IPF患者的肺内多条促纤维化途径被激活，许多抗纤维化途径被破坏。这项申请包括两项由研究人员和其他人的实验室提供的有希望的初步数据支持的建议，其中包括使用N-乙酰半胱氨酸(NAC)与甲磺酸伊马替尼(一种血小板衍生生长因子和转化生长因子β信号转导的抑制剂)或尿激酶(一种有效的纤溶剂)进行抗氧化治疗。通过过去和现在参与多中心IPF临床试验，以及他们建立和进行正在进行的由调查员发起的IPF试验，这项提案的研究人员清楚地表明，他们有能力招募所需的患者数量，以支持建立有效的临床IPF研究网络的拟议研究任务。南海湾临床研究网络将研究人员在基础科学和临床肺纤维化研究方面的积极经验与向南海湾独特人口结构提供临床肺纤维化试验的能力结合在一起。(摘要结束)

DESCRIPTION (provided by applicant): Idiopathic pulmonary fibrosis (IPF) is a debilitating and fatal disease for which there is no known cure. Recent epidemiologic studies have discerned that IPF is more common than previously recognized with an estimated 30,000 newly diagnosed cases in the United States yearly. There have been numerous expert reviews of late that have called our attention to the lack of evidence regarding the effectiveness of previously recommended treatment regimens that rely solely on the use of corticosteroids with or without other immunosuppressants for the treatment of IPF. It is now time to test the efficacy of novel approaches derived from decades of support from the NIH, ALA and private foundations for clinical and basic science lung fibrosis research. The overall goal of this proposal is to test the hypothesis that the most effective therapy for preventing disease progression will require multiple agents and is based on the knowledge that multiple profibrotic pathways are activated, and many antifibrotic pathways are disrupted in the lungs of patients with IPF. This application includes two proposals supported by promising preliminary data from the laboratories of the investigators and others, which involve anti-oxidant therapy using N-acetylcysteine (NAC) in combination with either imatinib mesylate (an inhibitor of platelet-derived growth factor and transforming growth factor beta signal transduction) or urokinase (a potent fibrinolytic). The investigators for this proposal have clearly demonstrated that they are capable of enrolling the number of patients required to support the proposed research mission of establishing an effective clinical IPF research network, through their past and current involvement in multi-center IPF clinical trials, as well as their establishment and conduct of an ongoing investigator-initiated IPF trial. The Gulf South Clinical Research Network combines the positive attributes of the investigators' experience in basic science and clinical lung fibrosis research with the ability to provide access to clinical IPF trials to the unique demographics of the Gulf South. (End of Abstract)