Two drugs offer hope for fatal lung disease

May 18, 2014 by Dennis Thompson, Healthday Reporter
2 drugs offer hope for fatal lung disease
Doctors have had little to offer patients with idiopathic pulmonary fibrosis.

(HealthDay)—A pair of drugs offers new hope to patients with a progressive, fatal disease that robs their breath by scarring the lungs, according to clinical trial results.

Both drugs, and nintedanib, appear to slow the advance of , or IPF, an incurable and previously untreatable disease that causes tissue deep in the lungs to stiffen and scar.

Patients with IPF have a three-year survival rate of 50 percent, worse than most forms of cancer, said Dr. Gary Hunninghake, a lung disease specialist at Brigham and Women's Hospital in Boston.

Few live longer than five years past diagnosis, and the only way to save their lives is to replace their lungs.

"The long and short is, this is a pretty big deal in the field of IPF," Hunninghake said. "It isn't curing the disease. This certainly isn't the end of therapy for these . But the idea there is something we can do besides referring them to lung transplant is a pretty big deal."

Results from the two drug trials are published in the May 18 online issue of the New England Journal of Medicine. The issue also reports on a third IPF drug called acetylcysteine that proved ineffective in a clinical trial.

More than 100,000 Americans suffer from IPF, said Dr. David Lederer, co-author of the pirfenidone study. Most are between 50 and 70 years old. The disease starts with shortness of breath or a dry, hacking cough, but soon robs the person's body of the oxygen needed to move about or properly function.

Doctors don't know what causes IPF, although they suspect that smoking, genetics, certain viral infections or acid reflux could play a role in damaging the lungs, according to the U.S. National Institutes of Health.

Of the two drugs, pirfenidone offers the most cause for hope among physicians and patients, said Dr. Gregory Cosgrove, chief medical officer for the Pulmonary Fibrosis Foundation.

Pirfenidone is approved for IPF treatment in other countries, but until now the U.S. Food and Drug Administration has denied approval based on mixed clinical trial results, Hunninghake said.

In this latest trial, doctors treated 555 IPF patients for one year with either pirfenidone or an inactive placebo. Pirfenidone produced a significant reduction in the rate of lung function decline, and improved patients' walking distance.

"This drug very clearly slowed down progression of the disease as measured by lung function, and seems to have an effect on mortality as well," said Lederer, co-director of the program at Columbia University Medical Center in New York City.

Researchers don't completely understand how pirfenidone works against IPF, Lederer said, but the drug seems to inhibit an important "growth factor" protein in the development of fibrosis. It also appears to have anti-inflammatory properties.

A second set of clinical trials tested the drug nintedanib against a placebo for one year in over 1,050 IPF patients.

Nintedanib also significantly reduced the rate of decline, improving patients' ability to breathe. The drug specifically targets the "growth factor" proteins that cause lung tissue to stiffen and scar.

Gastrointestinal problems including diarrhea were the most common side effects of both medications, researchers reported.

The matter of approval now rests with the FDA. "Both of these trials were designed more than likely to get FDA approval after their publication," Hunninghake said.

Overall, the results of these trials will provide some much-needed encouragement to IPF patients. "It is an optimistic time for patients with fibrosis," Cosgrove said.

The disease has been difficult to crack because animal models of lung fibrosis don't translate well to humans, he said. But the rapid advance of DNA sequencing has given researchers another avenue to understand how fibrosis works.

"It's been frustrating when we have not identified an effective therapy over the past 10 to 15 years," Cosgrove said. "But that degree of frustration has prompted the IPF community to really come together to support participation in , and those trials have provided a foundation for these new advances."

Explore further: Newly identified biomarkers help predict outcome in idiopathic pulmonary fibrosis

More information: For more information on idiopathic pulmonary fibrosis, visit the U.S. National Institutes of Health.

Related Stories

Newly identified biomarkers help predict outcome in idiopathic pulmonary fibrosis

October 2, 2013
A Yale-led study has identified a gene expression profile that can predict outcomes and lead to better treatment for one of the most lethal lung diseases, idiopathic pulmonary fibrosis (IPF). The study appears in Science ...

HRCT scans can identify deadly lung disease

February 17, 2014
People who have suspected idiopathic pulmonary fibrosis (IPF) without typical patterns on high resolution computed tomography scans could in future be spared the substantial risks of lung biopsy and be given a confident diagnosis ...

Delayed access to tertiary care associated with higher death rate from type of pulmonary fibrosis

July 1, 2011
Idiopathic pulmonary fibrosis (IPF)―scarring and thickening of the lungs from unknown causes―is the predominant condition leading to lung transplantation nationwide. Columbia University Medical Center researchers ...

Finding cellular causes of lung-hardening disease

September 17, 2013
(Medical Xpress)—Idiopathic Pulmonary Fibrosis, or IPF, is an incurable lung disease that, over time, turns healthy lung tissue into inflexible scar tissue – hardening the lungs and eventually causing respiratory distress ...

First, do no harm: Study finds danger in standard treatment for a serious lung disease

May 20, 2012
A combination of three drugs used worldwide as the standard of care for a serious lung disease puts patients in danger of death or hospitalization, and should not be used together to treat the disease, called idiopathic pulmonary ...

Recommended for you

Don't hold your nose and close your mouth when you sneeze, doctors warn

January 15, 2018
Pinching your nose while clamping your mouth shut to contain a forceful sneeze isn't a good idea, warn doctors in the journal BMJ Case Reports.

New antifungal provides hope in fight against superbugs

January 12, 2018
Microscopic yeast have been wreaking havoc in hospitals around the world—creeping into catheters, ventilator tubes, and IV lines—and causing deadly invasive infection. One culprit species, Candida auris, is resistant ...

Dengue takes low and slow approach to replication

January 11, 2018
A new study reveals how dengue virus manages to reproduce itself in an infected person without triggering the body's normal defenses. Duke researchers report that dengue pulls off this hoax by co-opting a specialized structure ...

Different strains of same bacteria trigger widely varying immune responses

January 11, 2018
Genetic differences between different strains of the same pathogenic bacterial species appear to result in widely varying immune system responses, according to new research published in PLOS Pathogens.

Human protein may aid neuron invasion by virus that causes hand, foot, and mouth disease

January 11, 2018
A human protein known as prohibitin may play a significant role in infection of the nervous system by EV71, one of several viruses that can cause hand, foot, and mouth disease. Issac Too of the National University of Singapore ...

Untangling how Epstein-Barr virus infects cells

January 11, 2018
A team led by scientists at Northwestern Medicine has discovered a new epithelial receptor for Epstein-Barr virus, according to a study published recently in Nature Microbiology.

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.