A new study published in the Journal of the American Medical Association (JAMA), has reported a new prevention treatment for individuals at high risk of developing colon cancer due to the APC (adenomatous polyposis coli) gene. Inheriting a mutation in the APC gene leads to a nearly 100% lifetime risk of developing colorectal cancer. This risk can be eliminated by removing the large intestine. However, these individuals also have up to a 15% risk of getting cancer in the small intestine, which is the leading cause of cancer death for these patients.
Familial adenomatous polyposis (FAP) is an inherited disorder that occurs in 1 out of 10,000 people. It is caused by a mutation in the APC gene that is inherited from one parent. Patients with FAP form hundreds to thousands of polyps in both their large and small intestine.
The current clinical trial tested a drug combination of sulindac (inhibitor of COX-2) and erlotinib (inhibitor of EGFR) to see if the development of polyps and cancer in the small intestine could be decreased. Ninety-two FAP patients were identified from the University of Utah Huntsman Cancer Institute Hereditary Gastrointestinal Cancer Registry. Half received the drug combination and were directly compared to the other half of FAP patients that were not treated. All patients received endoscopy before and after treatment to evaluate the size and number of polyps.
Individuals receiving the drug treatment were found to have developed significantly fewer and smaller polyps with an overall reduction in polyps of 71%.
While this preliminary trial is encouraging, longer follow up is required to better determine long-term outcomes and side effects.
Reference: Samadder N, Neklason D, Boucher K, et al. Effect of Sulindac and Erlotinib vs Placebo on Duodenal Neoplasia in Familial Adenomatous Polyposis. JAMA. 2016;315(12):1266-1275. doi:10.1001/jama.2016.2522. Retrieved from: http://jama.jamanetwork.com/article.aspx?articleid=2504814
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