Gulf South IPF Clinical Research Network

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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)