Inheriting a mutation in the APC gene leads to a nearly 100% lifetime
risk of colorectal cancer. While colon cancer can be kept at bay by
removing the large intestine, these patients also have up to a 15% risk
of getting cancer in the small intestine, which is the leading cause of
cancer death in this patient group. A new study published in the Journal of the American Medical Association (JAMA),
has identified the first prevention treatment for these patients, a
two-drug combination that significantly reduces the number and size of
precancerous polyps in the small intestine.
Deborah Neklason, PhD, a researcher on the study, says the current
clinical trial used the information obtained in basic science to test a
drug combination of sulindac (inhibitor of COX-2) and erlotinib
(inhibitor of EGFR). "This trial is an effort to go at two pathways that
intersect and see if we could drive down the development of polyps and
cancer in the small intestine," says Neklason.
Ninety-two FAP patients were identified from the Huntsman Cancer
Institute Hereditary Gastrointestinal Cancer Registry, one of the
largest registries in the world, and were entered into a trial where
half received drug and half placebo. The trial was blinded so neither
the patients nor researchers knew who was getting the drug. Both groups
of patients received an endoscopy before the trial began and again after
six months in order to visualize and characterize the size and number
of polyps before and after treatment.
At the time of the six-month endoscopy, the drug treatment group had
significantly fewer (p<0.001) and smaller polyps than the placebo
group with an overall reduction in polyps of 71% (p<0.001).
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