Mayo Clinic Researcher Finds Potential Target to Control Rare Genetic Disease That Strikes in Teen Years

A new study from researchers at Mayo Clinic Center for Individualized Medicine, described in the New England Journal of Medicine, reveals patients with the most severe FAP disease may benefit from a novel combination therapy. The study points to a possible target for intervention and prevention.

Author Niloy Jewel (Jewel) Samadder, M.D., a gastroenterologist and cancer genetics expert at Mayo Clinic’s campus in Ariz., and his colleagues, studied 171 patients with FAP who had various disease stages, at 20 centers in the U.S., Canada and Europe. Researchers evaluated the benefit and safety of the combination of eflornithine and sulindac compared to each drug alone. The investigation represents the largest international phase III FAP trial to date involving pharmaco-prevention — the use of a medication to prevent the need for surgery or other disease progression.