Mobile Menu Button
    • 12 DEC 14
    • 0

    Serendipitous Fruit-Fly Experiment Leads to Innovative LAM Treatment

    “Until the 1990s, almost nothing was known about Lymphangioleiomyomatosis (LAM). There were few clues about the origins of bizarre, destructive behaviors of the innocent-looking cells that infiltrated the lung, except that tuberous sclerosis patients seemed to be particularly vulnerable to develop the disease. Then, basic investigators in San Francisco discovered that tuberous sclerosis genes controlled cell size and growth in a series of breakthrough experiments in fruit-flies. The importance of tuberous sclerosis proteins in the regulation of the signaling protein mTOR and downstream cellular processes was rapidly dissected, and rodent studies were conducted that demonstrated feasibility and promise of mTOR inhibitors. A phase I/II trial at Cincinnati Children’s Hospital Medical Center showed that the drug shrunk kidney tumors, and also seemed to have a beneficial effect on lung function. A larger trial called MILES was designed at the University of Cincinnati, and executed at a dozen sites in the US, Canada and Japan. MILES demonstrated that therapy was effective. There is no better example of what can be accomplished when clues of nature are abundant, molecular targets are known, biological plausibility is high and patient and physician communities are highly motivated and well-organized.”

    Frank McCormack, MD, Director, Division of Pulmonary, Critical Care & Sleep Medicine

    Francis X. McCormack, MDFrancis X. McCormack, MD
    Director, Division of Pulmonary, Critical Care & Sleep Medicine
    Professor of Medicine, Department of Internal Medicine
    University of Cincinnati Medical Center
    Medical School: University of Texas Medical Branch

    Leave a reply →