A Small Molecule Therapeutic for the Treatment of Idiopathic Pulmonary Fibrosis
Project Abstract:
Fibrotic lung disease is a poorly understood heterogeneous syndrome affecting the lung interstitium Although there are many conditions associated with lung scarring idiopathic pulmonary fibrosis IPF is often considered a prototypical disorder Patients suffering from IPF experience an insidious progression of shortness of breath and 6070 of afflicted individuals die within 5 years of their diagnosis The two recently approved drugs for IPF only slow disease progression and cannot restore lost lung function The lack of therapies that halt the progression of IPF is a major unmet medical need Plasminogen activator inhibitor1 PAI1 regulates processes such as fibrinolysis and wound healing However PAI1 is associated with many disease processes including fibrosis of the lung kidney and heart and it is thought that PAI1 contributes directly to disease progression and that its inhibition may be an effective approach to treat a wide variety of fibrotic diseases This application will advance toward IND a small molecule PAI1 inhibitor with in vivo efficacy in murine models of IPF
