Temple Lung Center Offers Promising New Drug for Idiopathic Pulmonary Fibrosis

A new investigational drug that could be a game changer in the treatment of idiopathic pulmonary fibrosis (IPF) is being offered to patients at Temple Lung Center as part of a limited "expanded access" program in advance of the drug's regulatory approval.
 
Results of a recently published phase III trial showed that the drug, pirfenidone, slowed disease progression in patients with IPF, a debilitating and deadly lung condition. The need for new therapies for IPF is critical, given that the median time of survival for patients is two years from the time of diagnosis.
 
"Previously there wasn't evidence for effective drug therapy in patients with IPF ," said Gerard J. Criner, MD, Professor of Medicine at Temple University School of Medicine, and Director of the Temple Lung Center, a recognized national leader in clinical care and research for lung disease.
 
Pulmonary fibrosis, which is characterized by scarring in the lungs, is a progressive and irreversible condition. Tissue deep inside the lungs becomes thick, stiff and scarred, and in the process breathing becomes increasingly more difficult. Eventually patients have trouble breathing even at rest and must rely on oxygen therapy. Some younger patients get lung transplants, but most people with severe disease end up dying from it.
 
The disease is usually of unknown origin — thus the name "idiopathic" pulmonary fibrosis. According to the American Lung Association, about 140,000 Americans have the disease, which most commonly affects persons between the ages of 50 to 75.
 
"A better understanding of biology of IPF and the pathways involved in the disease has led to the development of new drug targets," said Dr. Criner, who is also Director of Pulmonary and Critical Care Medicine at Temple University Hospital.
 
Pirfenidone has not yet been approved by the federal Food and Drug Administration (FDA) for use in IPF patients, but Dr. Criner said that Temple has been selected by the drug manufacturer, InterMune, as an evaluation site during the pre-approval stage. Under the expanded access program for pirfenidone, all eligible IPF patients will receive the drug.

Temple has been involved in the investigation of several IPF drugs, and the results of studies were reported in the May 29, 2014 edition of the New England Journal of Medicine.

In the first study, researchers at Temple along with other NIH- sponsored investigators concluded that a drug commonly used to treat IPF — N-acetylcysteine (NAC) — offered no benefit over a placebo pill when it came to preserving lung function in IPF patients with mild-to-moderate lung function.
 
The second published study combined the results of two trials that tested the ability of nintedanib, developed by Boehringer Ingelheim, to reduce the decline in lung function in IPF patients as measured by forced vital capacity (FVC.) The drug performed better than a placebo when studied in a total of 1,066 patients over a 52-week period. The researchers said the reduction in the decline in FVC was "consistent with a slowing of disease progression." This agent will be evaluated further in an additional clinical trial that will soon be available at Temple.
 
The third study involved 555 IPF patients with mild-to-moderate disease who were assigned to take either the drug pirfenidone or a placebo pill. Researchers reported that the drug, as compared with placebo, reduced disease progression, "as reflected by lung function, exercise tolerance, and progression-free survival." The drug had gastrointestinal and skin side effects but in most cases subjects were able to continue the therapy despite these complaints. Pirfenidone is now available on a "compassionate use" basis at select medical centers, including Temple, as the drug's manufacturer awaits FDA review and approval.
 
IPF patients interested in learning more about any of the available Temple IPF research studies or the expanded access program for pirfenidone should call 215-707-8113 for more details about the studies or criteria for participation.

Editor's Note: Neither Dr. Criner nor any member of his immediate family has financial interest in InterMune or Boehringer Ingelheim.