LAM Foundation Research

The roadmap that The LAM Foundation has charted to find an effective treatment for our disease is simple: gene, protein, pathway, target, trials, therapy. The first step began in 1995 as the "Journey of a Thousand Miles", and 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 cellular pathway that was affected in tuberous sclerosis was also relevant to LAM and that LAM cells responded to 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. LAM Foundation research 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.

Click here for Principles that guide The LAM Foundation Research Program

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