Hope for patients with pulmonary fibrosis: In an international study, researchers at LMU University Hospital Munich have shown for the first time that an inhaled drug can significantly slow down the progression of the severe lung disease and improve quality of life at the same time. The already known drug treprostinil, previously used for pulmonary hypertension, proved to be effective and well tolerated .

The results, which have been published in the New England Journal of Medicine, mark an important step forward in the treatment of idiopathic pulmonary fibrosis - a disease that has been difficult to treat until now. Further studies will now clarify whether the new therapy concept will soon be approved.

In pulmonary fibrosis, damage to the alveoli (air sacs in the lungs) leads to defective healing with the formation of scarred areas and shrinkage of the lungs.

Due to the loss of alveoli, oxygen can no longer be adequately transferred to the blood, which initially leads to a lack of oxygen during exertion and later also at rest, and performance decreases further and further.

At the same time, the lung tissue becomes increasingly stiff due to the scarring, so that the work of breathing increases and the subjective feeling of breathlessness develops.

First of all, possible causes must be found or ruled out. These include certain organic or inorganic dusts (e.g. quartz dust, asbestos, mold-contaminated dusts in agriculture, etc.). Medication and rheumatic diseases can also trigger pulmonary fibrosis. In these cases, avoiding or treating the triggering cause is crucial.

If fibrosis has already occurred, there are two approved drugs - nintedanib and pirfenidone, which delay the progression of the disease and, in particular, lung shrinkage. A third drug with a slightly more favorable side effect profile - nerandomilast - has already been approved in the USA and is expected to be available in Germany in 2026.

In addition, pulmonary fibrosis patients benefit from general and symptomatic treatment measures such as oxygen administration if oxygen deficiency is already present, pneumological rehabilitation and moderate exercise, good nutrition with avoidance of obesity as well as of weight loss.

If all conservative treatment options have been exhausted and the disease is still progressing, lung transplantation remains the definitive treatment option. However, this requires that the patient is neither too healthy nor too ill and is therefore only an option for a small proportion of those affected.

Treprostinil is a prostacyclin analog that represents a novelty in the field of pulmonary fibrosis therapy due to its inhaled administration. Due to its effect on the pulmonary vascular system and the connective tissue cells, it can have a doubly beneficial effect on the disease process in pulmonary fibrosis. The study data now available are therefore particularly encouraging, as the shrinkage process has stabilized and the quality of life and, in particular, breathlessness have improved compared to the control group.

Treprostinil was originally developed for the treatment of pulmonary hypertension and is approved for both subcutaneous and intravenous administration in this indication and is used very successfully. In the so-called INCREASE study, inhaled treprostinil was tested in patients with pulmonary fibrosis and additional pulmonary hypertension with positive results, as a result of which the US Food and Drug Administration (FDA) approved inhaled treprostinil for this indication. In Europe, however, approval is still pending.

In individual cases, Treprostinil can be imported via the international pharmacy (internationale Apotheke) and administered as "off label use". However, the organizational effort and costs are very high. It also requires individual approval and confirmation of cost coverage by the respective health insurance company.

Currently, two large studies with inhaled treprostinil are still being conducted for the indication idiopathic pulmonary fibrosis (IPF) in the USA and Canada and for the indication progressive pulmonary fibrosis (worldwide). If these studies confirm the positive results of the first study, approval can be expected.