Researchers have identified a much-needed treatment for patients with a fatal lung condition.
Dr Sophie Fletcher led Southampton’s involvement in the global trial, recruiting patients with the rare lung disease idiopathic pulmonary fibrosis (IPF).
It is the first phase III trial success for this disease in ten years. The results offer new hope for these patients, who currently have very few options.
Short life expectancy
IPF is a rare disease that causes scarring of the lungs. Patients experience shortness of breath that gets worse over time, to the point that they may need oxygen.
Other symptoms include a dry and persistent cough, chest discomfort, fatigue and weakness.
People diagnosed with this condition only have a life expectancy of 3-5 years from diagnosis. It currently has limited treatment options, which can only be prescribed by a specialist centre.
Although considered rare, IPF affects approximately three million people worldwide. The disease primarily affects patients over the age of 50, and affects more men than women.
FIBRONEER™-IPF was the largest trial ever run for IPF, with over a thousand patients taking part in more than 30 countries. University Hospital Southampton (UHS) was one of over 330 sites involved.
IPF is one of the more common interstitial lung diseases (ILD). Prof Mark Jones leads the ILD service at UHS. He is also Professor of Respiratory Medicine at the University of Southampton.
“There is a high unmet need for patients with IPF,” he said. “Current approved treatments at best halve the rate of disease progression, and frequently patients have side effects that prevent treatment continuation.”
Trial success
The trial results suggest that the medicine nerandomilast, developed by the biopharmaceutical company Boehringer Ingelheim, slows down the decline in lung function in patients with IPF.
Patients with IPF who took part were randomly assigned to one of three groups. The first two groups both took a nerandomilast tablet twice a day, but they were different doses for each group. The third group took a placebo tablet, which looked the same but didn’t contain the active ingredient.
A Forced Vital Capacity test measured their lung function. This records the maximum amount of air you can forcibly breathe out from your lungs after taking the deepest breath you can.
The results showed that, after 52 weeks, patients taking nerandomilast had better lung function than those taking the placebo.
Dr Sophie Fletcher is a Consultant Respiratory Physician and part of the UHS Research Leaders Programme.
“The UHS Clinical Respiratory research team seeks out and delivers research trials that offer therapies to our patients, providing options where there are none,” she said, “benefiting both the participant and through the gain of knowledge guide improved patient care.
“As a research team, we are proud to be part of such a positive landmark trial.”
Getting it approved as a treatment
Boehringer Ingelheim are now seeking approval from the US Food and Drug Administration and other Health Authorities worldwide for nerandomilast to become a new IPF treatment.
The company are also conducting another trial of nerandomilast in patients with a second ILD, progressive pulmonary fibrosis (PPF).
“This is the first IPF phase-III-trial in a decade to meet its primary endpoint,” said Ioannis Sapountzis, Head of Global Therapeutic Areas at Boehringer Ingelheim.
“Today’s announcement represents the next step in our long history in the research of this disease. IPF has a high unmet need for patients, and we are continuously fostering our research activities to develop more options for one of the most common interstitial lung diseases.”
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