New Treatments for Deadly Idiopathic Pulmonary Fibrosis

New treatments are helping slow the deadly course of a rare lung disease that is on the rise in the U.S. and world-wide.

Idiopathic pulmonary fibrosis leads to a buildup of scar tissue that makes it increasingly difficult for the lungs to function normally and provide the body with oxygen. The disease has no known cause, setting it apart from related pulmonary diseases where doctors can identify specific irritants or exposures to toxins such as asbestos. Its symptoms—shortness of breath and persistent cough—resemble diseases like asthma and emphysema, often leading to missed or delayed diagnoses.

Now, with improved understanding of IPF and better diagnostic guidelines for catching it in earlier stages, researchers say the number of people afflicted is substantially higher than previously thought. According to the National Institutes of Health, it affects an estimated 200,000 Americans, mostly middle-aged and older adults. A study last year in the Lancet Respiratory Medicine found that prevalence among Medicare patients over 65 more than doubled between 2001 and 2011 and is increasing annually.

Historically more men have been diagnosed, but IPF in women is increasing. It kills about 40,000 patients each year, the same number that die of breast cancer. Only half survive more than two to three years from the initial diagnosis as their lungs stop working, from causes including respiratory failure and pneumonia. Lung transplants are one option for treatment, but can be done only for a limited number of patients.

Two new drugs have been shown to slow progression, and pharmaceutical companies are exploring potential new therapies that may offer improvement to current medicines. They are looking at combinations with other drugs and the use of an approved therapy for basal cell carcinoma, a skin cancer, as a potential treatment for IPF.

Researchers suspect the disease is linked to an exaggerated or uncontrolled healing response, possibly triggered by the immune system, which produces excessive scar tissue in the lungs and thickens the walls of the alveoli, or air sacs. Smoking is one of the most recognized risks. Exposure to wood or metal dust, viral or bacterial lung infections, and a history of acid reflux disease are possible culprits.

Charles Boetsch, a 69-year-old consultant, says he was always fit and active, so he knew something was wrong in 2011 when he began experiencing a dry cough and extreme shortness of breath. After multiple tests with different doctors, a high-resolution CT scan in 2013 showed he had IPF; he decided against an invasive lung biopsy for further confirmation. He had never heard of the disease and was stunned to learn there is no cure.

Mr. Boetsch, who lives in Palm Harbor, Fla., used to smoke but quit 35 years ago. In June 2014, he enrolled in a so-called expanded access program for patients without other treatment options to take the drug Esbriet free of charge, in advance of its FDA approval.

The drug is one of two newly recommended in guidelines released in July by the American Thoracic Society, whose member physicians treat respiratory diseases and related illness. The drugs block processes involved in the scarring of lung tissue. They were approved by the Food and Drug Administration last fall, with the caveats that side effects and costs must be considered. The drugs run as much as $94,000 annually. While they don’t cure the disease, they have been shown to significantly reduce the decline in what is known as forced vital capacity, which is the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible.

Makers of the so-called antifibrotic drugs—pirfenidone, sold as Esbriet by Genentech, a unit of Roche Group, and nintedanib, sold as Ofev by German pharmaceutical company Boehringer-Ingelheim—offer support including free medicine to eligible patients who don’t have insurance, and copay assistance to those who qualify.

Mr. Boetsch suffered side effects including gastrointestinal disturbances, a metallic taste from the nine pills he had to take daily and fatigue. “There are ways to manage yourself through all of these side effects, but the mental aspect is the toughest, because it doesn’t leave your mind, ever, and you always wonder when the next shoe is going to drop,” he says.

Mr. Boetsch says he is encouraged that his lung function hasn’t declined since he started Esbriet. He is participating in another clinical trial. He walks and exercises daily. He eats a healthy diet, steering clear of refined sugar and dairy products and buying organic foods. An over-the-counter medication helps minimize mucus in his lungs. He still gets winded climbing stairs, but he and his wife, Robyn, have continued to travel. Last October they visited Italy and in June, took a trip to Alaska. He chose not to take an oxygen pack, and while he wasn’t able to hike vigorously, he says he might in the future.

Mr. Boetsch says friends who see him looking healthy sometimes forget he has the disease, and don’t understand that he gets tired and can’t participate in social situations as much as in the past. While he knows his treatment isn’t a cure, “it buys you more time,” he says, and he has chosen to ignore the dire mortality statistics. “If you can tolerate the drugs and have a favorable response, you may get twice to three times longer with a reasonable quality of life.”

The new guidelines for treating IPF also recommend strongly against traditional treatments that studies have shown can do more harm than good. They include the use of the blood thinner warfarin and a three-drug regimen including the steroid prednisone. “The evidence showed that the standard of care that has been used for decades is wrong,” says Ganesh Raghu, lead author of the guidelines and director of the Center for Interstitial Lung Disease at the University of Washington, in Seattle. Dr. Raghu has received consulting fees from drug makers but says specific treatment recommendations were made by nonconflicted panel members.

Dr. Raghu says more data is needed on the role of factors such as heredity.

In as many as 15% of cases, patients have another family member with IPF. One study by National Jewish Health, Duke University and Vanderbilt University seeks to identify a group of genes that predispose individuals to develop pulmonary fibrosis. Some patients remain in stable condition for extended periods, while others have a rapid progression or fluctuate between periods of stability and worsening symptoms, according to the nonprofit Pulmonary Fibrosis Foundation.

“Idiopathic pulmonary fibrosis is usually a progressive disease, but its course varies widely among individual patients,” says Teng Moua, a physician in the division of pulmonary and critical care at the Mayo Clinic in Rochester, Minn. “As the disease has become more well-known, we are finding it earlier and making attempts to slow it down and manage it.” A lung biopsy is sometimes used, but many cases are now confirmed with high-resolution CT scans. Dr. Moua prescribes his patients either Esbriet or Ofev after discussions about side effects and tolerance. Both appear to have similar effectiveness, but further research is needed “in determining whether they will be helpful long-term,” he says.