An already approved transplant-rejection drug is the first treatment to show a benefit for women with a rare lung disease that has had no cure or, until now, even a treatment.
The drug, sirolimus (Rapamycin), improved both lung function and quality of life in women with lymphangioleiomyomatosis, or LAM, according to a study published online March 16 in the New England Journal of Medicine.
“To have a therapy for this disease is rare and special,” said the study’s lead author, Dr. Francis X. McCormack, a professor and director of pulmonary, critical care and sleep medicine at the University of Cincinnati and scientific director of the LAM Foundation in Cincinnati, which helped fund the trial and also recruited participants.
LAM is characterized by progressive loss of lung function from the invasion of abnormal muscle tissue that obstructs airways. According to the American Lung Association, as many as 250,000 women worldwide might have the disease.