The LAM Foundation - LAM Research

Poster Session

The LAM Foundation began in 1995 with a "Journey of a Thousand Miles". The roadmap that we have charted for our journey to find an effective treatment for LAM is simple: gene, protein, pathway, target, trials, and therapy. The Foundation began funding research in 1996 and the first step was achieved in 2000 with Dr. Elizabeth Henske's discovery of the importance of tuberous sclerosis genes in LAM. Dr. Vera Krymskaya then showed us in 2003 that the TSC cellular pathway was dyregulated in LAM cells from patients and that LAM cell growth was inhibited by rapamycin. Drs. Ray Yeung and David Kwiatkowski demonstrated that tumors in mice and rats with tuberous sclerosis shrunk on rapamycin, and based on his laboratory data, Dr. Richard Lamb from England first uttered the heretical suggestion that we might treat LAM patients with rapamycin. The discoveries of these LAM Foundation-funded scientists, and those of countless other partners in the tuberous sclerosis and Drosophila biology fields, has now brought our destination into focus.

Promising molecular targets that are ready for testing in clinical trials have been identified, many of which are addressable with drugs that are FDA-approved for other indications. Research funded by The LAM Foundation is therefore focused on the development and validation of drug targets, prioritization of compounds for testing, and facilitation and implementation of phase I /phase II clinical trials.