Grant: Project Grant

Progressive lung fibrosis (scarring) is a devastating condition with poor prognosis and low quality of life. It can arise due to unknown causes (idiopathic) or due to scleroderma, an autoimmune disease characterized excessive scarring of lung and other organs. There are limited treatment options for patients diagnosed with idiopathic or scleroderma-related lung fibrosis. We have recently developed a technology based on a small peptides (small proteins) which are highly specific and display potent anti-fibrotic properties in vitro using scleroderma patient cells and in vivo using mouse models of lung and skin fibrosis. Our findings provide compelling reasons to advance our technology towards the development of a clinically effective product for the treatment of idiopathic and scleroderma lung fibrosis. Our technology is protected by two granted US patents, an approved European Union patent, a pending Canadian patent, with new patents in preparation for filing. The proposed research in the current application on the preclinical development of a candidate peptide is warranted to establish efficacy, dose range, pharmacokinetics, pharmacodynamics, safety and mode of delivery. Another goal is to develop a formulation for the delivery of the drug and testing its efficacy in animal models. These studies are expected to form the basis for future human clinical trials. The proposed research thus has the potential for the development of a novel anti-fibrotic agent for the treatment of patients with lung fibrosis.