Study Shows Rapamycin Reverses the Progression of the Fatal Lung Disease Lymphangioleiomyomatosis

Lymphangioleiomyomatosis (LAM) is an uncommon but extremely serious lung disease that typically targets females. It causes tumor suppressing signals to shut down, triggering an explosion of cell growth. Cysts are forms as smooth muscle cells get into the lungs. Breathing is hampered and abdominal tumors may form. Women experience shortness of breath, and their lungs may collapse.  Respiratory failure often occurs, with a lung transplant being the only real option for those in this situation. With little to no understanding as to the cause of the disease, therapy options were limited and not nearly substantial enough. Now a groundbreaking study has changed all that with the promising drug rapamycin.

The University of Cincinnati headed the Multicenter International LAM Efficacy of Sirolimus (MILES) trial. According to the Science Daily, the United States, Canada and Japan were all used as locations in the trial. Funding came from sources including the National Institutes of Health. The drug rapamycin, or sirolimus, was given for one year to the clinical group while a placebo was given to the second group. The sample included 89 patients with LAM who were 18 or older and had a confirmed diagnosis of the disease. They got either oral sirolimus of 2 miligrams daily or a placebo. The study was performed double-blind and randomly assigned to preserve integrity.

According to the Health News Digest, the results were staggering. Lung function tests were significantly better on the control group as compared to the placebo group. Lung function was stabilized, and quality of life was increased. In the second year of the trial, all medication was stopped, and the patients were observed. Unfortunately, a worsening of the control group was shown, indicating that continued use of the drug would be necessitated for long-term effects. Not everyone responded to the drug, however the results were still quite significant.

Rapamycin is typically used to help transplant patients better accept new organs and not reject them, but this study shows a large promise for the sufferers of lymphangioleiomyomatosis. It works by blocking a cell-signaling pathway to try to stifle the tremendous growth of cells that is so damaging.

The findings of the study were published in the online New England Journal of Medicine on March 16, 2011. Bruce Trapnell, MD, headed it. Frank McCormack, MD, was the lead investigator. Dr. Mark Brantlet, co-authored the paper. He works as a University of Florida professor, where some of the trials took place.     

Rapamycin is showing new hope to scores of women who suffer from lymphangioleiomyomatosis. New studies will occur in the future to hopefully create an effective treatment for the women who suffer from this disease.