Idiopathic pulmonary fibrosis (IPF) is still an incurable disease. Lung tissue becomes progressively scarred, and this process can only be slowed to a limited extent. The underlying cause remains only partly understood. Current standard therapy can slow the progression but cannot stop it. Many patients lose significant lung function and quality of life over the years.
This is why clinical research in this field is so important: every new option that measurably affects disease progression has a direct impact on patient care. A research team led by senior author Prof. Jürgen Behr, DZL researcher at the CPC-M site and Head of the Department of Medicine V at LMU University Hospital Munich, has now presented promising data on the treatment of idiopathic pulmonary fibrosis (IPF). The findings were published in the highly respected New England Journal of Medicine. Results from the phase‑3 trial show that the inhaled drug treprostinil slows the decline in lung function and reduces clinical worsening.
The study was large and methodologically clear: around 600 patients were randomized and treated double‑blind with treprostinil or placebo over 52 weeks. The study population was typical for the disease—older, predominantly male, and most were already on antifibrotic background therapy. This means the results reflect real‑world clinical practice.
The primary endpoint was the change in forced vital capacity (FVC) after 52 weeks. With treprostinil, median FVC declined by about 50 milliliters; in the placebo group, the decline was around 136 milliliters. Clinical worsening was also less frequent under treprostinil. The effect is therefore visible both in measurable functional data and in the course of the disease. The most common side effect was cough, reported by almost half of the patients receiving treprostinil. Notably, this is the only study to date that demonstrates a statistically significant and clinically relevant benefit in health‑related quality of life for patients in the active treatment arm.
For Prof. Behr, the translational aspect is key: “We have been working for years to bring new therapies from the laboratory into patient care. The study shows that this inhaled drug can indeed slow the progression of IPF. Treprostinil’s mechanism at the interface between pulmonary vessels and alveoli may also open up new therapeutic avenues. This is an important step, because until now we have only been able to influence the disease to a limited degree.” The inhaled route of administration is a deliberate choice. Treprostinil acts directly in the lungs and unfolds its potential antifibrotic effects in the affected tissue. Seeing this mechanism confirmed in a large clinical trial is a major success for the participating centers.
The results show that therapeutic progress in IPF is possible—although there remains a clear need for well‑tolerated and more effective treatments. For the research teams, the study provides momentum: it confirms that closely connected preclinical and clinical research can lead to tangible improvements for patients.
Originalpublikation: Nathan SD, Smith P, Deng C, De Salvo M, Wuyts W, Pavie-Gallegos J, Song JW, Kramer MR, King CS, Mackintosh JA, Chambers D, Miranda GV, Breytenbach N, Peterson L, Bell H, Flaherty KR, Behr J, Cottin V; TETON-2 Trial Investigators. Inhaled Treprostinil for Idiopathic Pulmonary Fibrosis. N Engl J Med. 2026 Mar 11. doi: 10.1056/NEJMoa2512911. Epub ahead of print. PMID: 41812190.
