Searching for a LAM Treatment

The LAM Foundation has been working with urgency to find an effective treatment for lymphangioleiomyomatosis (LAM). In the first five years of funding LAM research, LAM Foundation scientists reported three major discoveries, including evidence that LAM is genetic, the identification of a LAM gene, and a molecular explanation for abnormal smooth muscle cell growth in LAM. This data resulted in the identification of a potential experimental therapy called sirolimus, or rapamycin. The 2003 Cincinnati Rapamycin Trial was conducted at Cincinnati Children's Hospital and the University of Cincinnati. The results from that study, published in the New England Journal of Medicine on January 10, 2008, revealed that sirolimus reduced the size of angiomyolipomas and improved lung function in some patients with LAM. The number of patients were too small to determine if the beneficial effects of the drug outweighed the side effects and other risks. The conclusion was that sirolimus should be further tested in the treatment of LAM, tuberous sclerosis, and angiomyolipomas (kidney tumors) in larger trials.

Well-conducted clinical trials are the fastest and safest way to find improved treatments and preventions for diseases like LAM. Please become well-informed about the clinical trials that are available for LAM and consider enrolling.

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