01:23pm Tuesday 22 October 2019

New Treatment for Fatal Lung Disease Proves Promising

The results were published online May 14 in the prestigious British medical journal Lancet.

Researchers at 10 medical centers worldwide, including the Miller School, conducted two concurrent, nearly identical trials with close to 800 patients. As part of the CAPACITY (Clinical Studies Assessing Pirfenidone in IPF: Research of Efficacy and Safety Outcomes) program the patients were randomized to blinded treatment with oral pirfenidone or placebo for a minimum of 72 weeks. Marilyn Glassberg, M.D., associate professor of medicine, served as a principal investigator for the Miller School and was one of the study’s authors.

Pirfenidone, a novel antifibrotic and anti-inflammatory agent, is an experimental treatment that had shown promise in reducing decline in lung function in patients with idiopathic pulmonary function in an earlier Phase II study in Japan. The findings of that study led to regulatory approval of pirfenidone for the treatment of idiopathic pulmonary fibrosis in Japan.

“We have shown that pirfenidone could be a therapy for patients with idiopathic pulmonary fibrosis that is safe and could provide an initial standard of care,” said Glassberg. “Pirfenidone had a clinically meaningful effect on lung function in these patients with an excellent safety profile. The effect of the highest dose was seen as early as week 24 in this 72 week study.”

Idiopathic pulmonary fibrosis is characterized by progressive shortness of breath and irreversible loss of lung function. The median survival after diagnosis is two to five years.

“Since there are no approved therapies for patients with this devastating disease, the need to study pirfenidone as well as other therapies is of utmost importance,” Glassberg added. “There is a large unmet therapeutic need. With the recent publication of the isolation of human lung stem cells, and the ongoing studies on mesenchymal stem cells at the Miller School’s Interdisciplinary Stem Cell Institute, we are hopeful that new treatments are on the horizon for these patients.”

The study was funded by InterMune, Inc., which manufactures pirfenidone.

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