Molgramostim Shows Promise in Treating Autoimmune Pulmonary Alveolar Proteinosis
Inhaled molgramostim demonstrates significant improvements in lung function for adults with autoimmune pulmonary alveolar proteinosis, offering new hope for treatment. Read the latest research findings.
A recent study published in the August 2025 issue of the New England Journal of Medicine highlights the potential benefits of inhaled molgramostim, a recombinant human granulocyte-macrophage colony-stimulating factor, for adults diagnosed with autoimmune pulmonary alveolar proteinosis (aPAP). This condition, characterized by the accumulation of surfactant in the alveoli, impairs gas exchange and can lead to progressive respiratory issues.
In the randomized, double-blind phase 3 trial, 164 participants received either daily inhalations of molgramostim at a dose of 300 μg or a placebo over a span of 48 weeks. The findings demonstrated that those treated with molgramostim experienced a significantly greater improvement in pulmonary gas transfer capacity, measured by the diffusing capacity of the lungs for carbon monoxide (DLco). Specifically, the increase in DLco at 24 weeks was 9.8 percentage points with molgramostim compared to 3.8 points with placebo, with benefits maintained at 48 weeks.
Additionally, patient-reported outcomes using the St. George’s Respiratory Questionnaire showed an average score reduction of -11.5 points in the molgramostim group versus -4.9 in the placebo group, indicating better respiratory health and quality of life. Importantly, both groups exhibited similar rates of adverse and serious adverse events, underscoring the safety profile of inhaled molgramostim.
The study's lead author, Dr. Bruce C. Trapnell from Cincinnati Children’s Hospital Medical Center, emphasized that daily inhalation of molgramostim effectively improved lung function in aPAP patients, with sustained benefits over the treatment period. These promising results suggest that molgramostim could become a valuable therapeutic option for managing aPAP, providing patients with tangible improvements in their respiratory capabilities.
For more details, the full study can be accessed in the New England Journal of Medicine: [DOI: 10.1056/NEJMoa2410542].
source: https://medicalxpress.com/news/2025-08-molgramostim-beneficial-autoimmune-pulmonary-alveolar.html
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