In 1994 Sue Byrnes’s daughter was diagnosed with lymphangioleiomyomatosis (LAM), a chronic, progressive and life-limiting lung disease that strikes down women in their prime. At the time, no one was looking for a cure. Byrnes established The LAM Foundation to support research into this heart-breaking condition.
Using LAM cells derived from tissues procured by NDRI, researchers at the University of Pennsylvania conducted preclinical testing of the drug sirolimus. Results from these early studies were promising and eventually led to a clinical trial, which found that the drug stabilized lung function and improved quality of life in patients with moderate to severe LAM. In May 2015, the federal Food and Drug Administration approved sirolimus for use in patients with LAM — the first effective treatment for the disease.