For the first time, researchers have found drugs that can slow the decline of people with a fatal lung disease, idiopathic pulmonary fibrosis.
Studies of two experimental drugs were published in The New England Journal of Medicine and presented on Sunday in San Diego at a meeting of the American Thoracic Society. The drugs did not make patients better, but slowed the rate at which lung function got worse.
The odds of death increase as lung function decreases, so researchers hope that by stabilizing it, the drugs will prolong survival. But it is too soon to tell.
The disease causes scarring of the lungs, making it harder and harder to breathe. At least 80,000 Americans have it. Half of patients die in three to five years.
An editorial accompanying the studies called the results “a major breakthrough for patients,” but also cautioned that questions remained about how long the drugs would work and whether they would help people with severe disease.
Until now, there had been no treatments that could do anything other than relieve symptoms like coughing.
“There’s been nothing,” said Dr. Gary M. Hunninghake, the author of the editorial and a lung specialist at Brigham and Women’s Hospital in Boston. “That’s about to change. I think for a lot of patients this is going to be pretty exciting.”
Lung transplants save some patients with the disease, but many are too old or too ill for the surgery. What causes the lung scarring is unknown. Smokers and former smokers are at higher risk, but most people who smoke do not develop the disease, and many patients who do get it never smoked.
“Once you’ve been given the diagnosis, people start to plan for how long they’ll be living,” said Dr. Kevin K. Brown, an author of one of the studies and a lung specialist at National Jewish Health, a respiratory hospital in Denver. He added, “Patients hope they can get better, but pray they don’t get any worse.”
Neither of the drugs has been approved by the Food and Drug Administration. The data from the studies will be submitted to the agency as part of the process of seeking approval for the drugs.
One drug, pirfenidone, is made by InterMune, which is based in Brisbane, Calif. It has already been approved in Europe, Canada and Japan. InterMune paid for the study. The drug fights the scarring process, but how it does so is not fully understood.
The other drug, nintedanib, is made by the German company Boehringer Ingelheim, which helped pay for the study. Dr. Brown, who took part in that study, said the drug was originally being developed to treat cancer, and was tested with the lung disease when researchers learned that lung fibrosis had some biological features in common with cancer.
The drugs were studied separately and were not used in combination. Each was compared with a placebo, not with the other drug, so researchers say it is not clear whether one drug is better than the other.
Each drug was studied for a year, and one of the most important results was lung function, measured by breathing tests. Patients taking either drug had significantly smaller drops in lung function than those taking placebos. Both drugs had side effects, mostly not severe: In some patients, nintedanib caused diarrhea, and pirfenidone caused rashes and nausea.
Ed Duncan, 78, of Aurora, Colo., said he had been taking pirfenidone in various studies since 2007 or 2008. He has had idiopathic pulmonary fibrosis for 10 years, and suspects that his long survival with the disease may be due at least in part to the drug. He needs oxygen all the time, but can get about, slowly. Strenuous exercise is out of the question.
“Ten years was my bucket list,” Mr. Duncan said. “I was happy to make that. The way I feel now it could be several more years.”
InterMune announced on Friday that it would make pirfenidone available for free to some patients in the United States through an “expanded access program,” designed to help patients obtain experimental drugs that have not yet been approved. The move seems most likely to give InterMune a head start since patients will probably not have access to the other drug until it is approved.
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