New Treatment Tested at UTHSCT Proves Successful

TYLER — A new treatment that was tested at The University of Texas Health Science Center at Tyler has proven to be successful against a rare but serious lung disease called LAM, short for lymphangioleiomyomatosis. �Finally, a drug � sirolimus � has been found that can slow the progression of LAM, an unusual disorder that can lead to serious disability and death,� said James Stocks, M.D., a nationally known lung disease specialist. �LAM primarily strikes younger women who often still have young children at home. Though this is not a miracle cure, it is an important first step,� added Dr. Stocks, the principal investigator of the LAM clinical study at UTHSCT. Most women with the disease live only another eight to 10 years after their diagnosis.

Sirolimus, also known as rapamycin, is the first drug to effectively treat LAM, Dr. Stocks said. Because it suppresses the body�s immune system, sirolimus currently is used to prevent rejection of transplanted organs. It also is used as a coating for cardiac artery stents � metal mesh tubes that prop the arteries open � to prevent clotting. During one year of treatment, sirolimus improved the lung function and quality of life of the women who took it, according to the Multicenter International LAM Efficacy of Sirolimus (MILES). MILES is the first randomized, controlled study of a treatment for LAM. In this study, women were randomly assigned to receive sirolimus or a placebo, which is a substance that has no effect, such as a sugar pill.

In women with the disease, abnormal, out-of-control, muscle-like cells take over their lungs, lymph nodes, and kidneys. These mutant cells destroy normal lung tissue, making breathing difficult, according to the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health (NIH). Sometimes the lungs of women with LAM collapse, causing pain and shortness of breath. LAM usually worsens over time, but how quickly it progresses varies greatly, the NHLBI says. Symptoms of LAM are shortness of breath, especially during physical activity; chest pain; frequent cough; and wheezing. The results of MILES were published in a recent online edition of The New England Journal of Medicine.

Besides UTHSCT, other institutions participating in the study included National Jewish Health in Denver; Cincinnati Children�s Hospital Medical Center; the Cleveland Clinic Foundation; the University of California at Los Angeles; Brigham and Women�s Hospital in Boston; the University of Toronto, Canada; and the National Hospital Organization Kinki-Chou Chest Medical Center in Osaka, Japan.

Funding for the study was provided by the NIH Office of Rare Disease Research; the Food and Drug Administration; the LAM Foundation, the Japanese Ministry of Health, Labor, and Welfare; the Canadian Institutes of Health Research; Cincinnati Children�s Hospital Medical Center; the University of Cincinnati; the Tuberous Sclerosis Alliance Rothberg Courage Award; Vi and John Adler; and the Adler Foundation.