People who live with autoimmune pulmonary alveolar proteinosis (aPAP), a disease that causes the lung’s tiny air sacs (alveoli) to fill up with an oily substance called surfactant normally present in small amounts, are forced to undergo anesthesia and get their lungs washed out every year or so just to survive.
But an international team of researchers reports in the New England Journal of Medicine dramatically transforming aPAP treatment by helping develop a drug that patients can inhale once a day so their body will clear out the pulmonary waste.
Led by Cincinnati Children’s pulmonary physician-scientist, Bruce Trapnell, MD, the research group published their findings on Sept. 7, 2020, while presenting study data at the European Respiratory Society International Congress, conducted virtually this year because of COVID-19.
Physicians and scientists from 34 institutions in 18 countries, conducted a double-blind, placebo-controlled, clinical trial to evaluate an immuno-stimulator drug called molgramostim. The drug is a recombinant form of the protein GM-CSF (granulating macrophage colony stimulating factor). The study involved 138 people.
The inhaled drug was administered once daily, which resulted in improved respiratory function and overall health status, the co-authors report.
“No drugs have been approved for treating PAP in any country, so this one could radically improve the lives of people with aPAP,” Trapnell says. “This could reduce or eliminate the need for whole-lung lavage, which is an invasive and inefficient procedure done under general anesthesia. One lung is physically washed out with saline while the other is mechanically ventilated.”
This disease strikes an estimated seven to 26 people per 1 million population, or roughly 2,000 to 8,000 people in the United States. The lavage treatment has been the only way to extend their lives.