(CINCINNATI) -- The drug sirolimus (Rapamycin) is the first to produce any benefit for women who suffer from a rare lung disease called lyphangioleiomyomatosis, or LAM. The disease has had no cure, and until now, no suitable treatments.
While sirolimus has already been approved as a transplant-rejection drug, a study released Wednesday in the New England Journal of Medicine reports that when given to LAM patients, lung function and quality of life is improved.
LAM is the loss of lung function due to the abnormal growth of muscle tissue causing airway obstruction. Internationally, an estimated 250,000 women are undiagnosed or misdiagnosed, according to the LAM Foundation.
The study's lead author, Dr. Francis X. McCormack of the University of Cincinnati and scientific director of the LAM Foundation, noted the "rare and special" nature of the treatment's discovery, but said that stabilized lung function only occurred "for as long as patients took the drug." If a patient stopped taking the drug, a decline in lung function would resume, McCormack said.
McCormack added that sirolimus costs around $8 a tablet, and that the recommended dosage is two tablets a day.
Jill Raleigh, executive director of the LAM Foundation, indicated the treatment isn't likely to be a cure-all for everyone.
"It's not a cure," she said. "But it's hope."
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