MicroRNA Mimic Shown to Reverse Effects of Pulmonary Fibrosis

A potential new treatment that reverses the effects of pulmonary fibrosis, a respiratory disease in which scars develop in the lungs and greatly suppress breathing, is being assessed by scientists. The therapy uses a microRNA mimic, miR-29, which is delivered to lung tissue intravenously. In lab mice, miR-29 not only blocked pulmonary fibrosis, it reversed fibrosis after several days.

The findings were published September 19, 2014, in the journal EMBO Molecular Medicine. “The mimic, when injected into the blood, goes to the lung and it has a sustained effect. We are very impressed that it can reverse fibrosis, not only prevent it,” said Naftali Kaminski, MD, a professor at the Yale School of Medicine (New Haven, CT, USA), and section chief of pulmonary, critical care, and sleep medicine, and corresponding author of the study.

The research is a collaboration between Yale and miRagen Therapeutics Boulder, CO, USA), a pharmaceutical company. The company had developed miR-29 earlier as a possible therapy for cardiac disease. Dr. Kaminski, whose group innovated research in microRNA in lung fibrosis, visualized the potential for use of miR-29 in pulmonary fibrosis, as did Dr. Eva van Rooij, the scientist who discovered the role for miR-29 in cardiac fibrosis, and is a senior coauthor on the study. “I’m particularly excited about working with this microRNA,” said Dr. van Rooij. “All evidence points to it being a master regulator of fibrosis.”

The next phase of the study, according to Dr. Kaminski, will be to begin evaluating miR-29 as a therapeutic for human idiopathic pulmonary fibrosis (IPF). Once considered a rare disease, IPF now affects more than 200,000 people in the United States, where approximately 30,000 people die from IPF every year. The median survival from diagnosis is three to five years, and in spite of recent potential developments, there is no intervention that reverses the disease.

Related Links:
Yale School of Medicine
miRagen Therapeutics