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New research demonstrates potential diagnosis, treatment benefits
Date:1/10/2008

Cincinnati, OH (January 10, 2008) Studies published in the Jan. 10 edition of the New England Journal of Medicine (NEJM) are providing clues into the treatment and diagnosis of LAM, or lymphangioleiomyomatosis, a progressive and deadly lung disease that affects women in their childbearing years. There currently are no treatments for LAM and scientists estimate as many as 250,000 women may be going misdiagnosed or undiagnosed.

Researchers from Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine reported on a study testing the drug sirolimus in patients with LAM or tuberous sclerosis complex (TSC) with angiomyolipomas, benign kidney tumors common to both diseases. Approved to help transplant patients fight organ rejection, sirolimus treatment resulted in a 50 percent reduction in tumor growth; a significant improvement in lung function was observed in LAM patients. In addition, a letter published in the same issue of NEJM reports on preliminary data to support the use of a serum marker test to confirm a diagnosis of LAM. The disease has traditionally required a lung biopsy or CT scan for confirmation of diagnosis, contributing to diagnosis complications.

These studies represent significant advances for LAM patients, said Leslie Sullivan-Stacey, J.D., President and CEO of The LAM Foundation, a supporter of both studies. The LAM Foundation has been the driving force behind major breakthroughs in LAM research over just the last decade, and we now have scientific evidence to support further study of treatments and diagnostic tools. The sirolimus study already is serving as the basis for other studies in TSC and LAM, including the first-ever LAM treatment trial, now enrolling patients.

In addition to these U.S. studies, a second letter to the editor from researchers in the United Kingdom reports on a Phase II study of sirolimus in patients with TSC and sporadic LAM. An editorial authored
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Contact: Shellie Byrum
sbyrum@spectrumscience.com
202-955-6222 x2516
The LAM Foundation
Source:Eurekalert

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