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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.  

Cancer Prevention Pharmaceuticals, Inc. (CPP), a private biotech company developing novel therapeutics to prevent cancer and other diseases, today announced The New England Journal of Medicine (NEJM) has published results from its landmark FAP-310 Phase 3 clinical trial of CPP-1X/sul as a pharmaco-preventive treatment for adults with familial adenomatous polyposis (FAP). FAP is a rare genetic disease that if left untreated progresses to colorectal cancer in nearly 100% of patients. Standard of care is surgery as there are no approved drugs for FAP. The FAP-310 Phase 3 trial compared the performance of the combination of CPP-1X (eflornithine) and sulindac (CPP-1X/sul), to each single agent alone in delaying progression to an FAP-related clinical event, such as surgery. The trial enrolled 171 patients and was conducted over many years where patients received daily treatment for 2-4 years. It was the largest prospective controlled study ever performed in FAP, and the only clinical event driven trial ever conducted.