CINCINNATIResearchers at the University of Cincinnati (UC) and Cincinnati Children's Hospital Medical Center have found that the FDA-approved drug sirolimus, used primarily to prevent rejection in organ transplant patients, stabilized lung function in women with lymphangioleiomyomatosis (LAM).
The Multicenter International LAM Efficacy of Sirolimus (MILES) trial was the first randomized, controlled study designed to develop a therapy for this life-threatening illness, which currently has no cure or treatment.
These results are being reported in the March 16, 2011, online edition of the New England Journal of Medicine.
LAM is a progressive, cystic lung disease that occurs almost exclusively in women. In LAM, an unusual type of smooth muscle cell grows uncontrollably and spreads from an unknown source to restricted areas in the body, including the lungs, lymph nodes and vessels and kidneys, limiting the flow of air, blood and lymph.
Shortness of breath and recurrent lung collapse are common in patients with LAM, and until now, lung transplantation has been the only hope for patients who progress to respiratory failure.
"LAM affects about five people per million and occurs in 30 to 40 percent of women with tuberous sclerosis complex (TSC), a genetic disorder which also causes tumors to form in the kidneys, brain, heart and other organs," says Frank McCormack, MD, director of the pulmonary, critical care and sleep medicine division at UC and lead investigator on the study.
"Sirolimus, otherwise known as rapamycin, showed promise for patients with LAM in a pilot study conducted in Cincinnati and reported in the New England Journal of Medicine in 2008, but the relative risks and benefits of sirolimus for treatment of patients with this condition have remained unclear. In this international, multicenter study, we evaluated the safety and efficacy of one year of sirolimus in stabilizing and/or improv
|Contact: Katie Pence|
University of Cincinnati Academic Health Center