Familial Adenomatous Polyposis (FAP) is a rare colorectal cancer syndrome attributed to a mutation in the Adenomatous Polyposis Coli (APC) gene. FAP is characterized by an abnormally high rate of cell division/growth in the epithelial cells lining the colon. These growths are referred to as colon polyps. Onset of the disease typically occurs in the teenage years with over 80% of patients forming polyps in the stomach and small intestines as well. The majority of patients with FAP, about 70%, inherit the APC gene mutation from one of their parents while the remaining patient population occurs through spontaneous mutation of the APC gene. Certain diagnostic tests, via a blood sample, can be performed to confirm the genetic mutation and determine the best course of treatment. Currently, surgical removal of the colon is the only effective treatment.
Additional information regarding FAP can be found on the NIH websites:
- http://ghr.nlm.nih.gov/condition/familial-adenomatous-polyposis
- http://rarediseases.info.nih.gov/GARD/Condition/6408/Familial_adenomatous_polyposis.aspx
Marina Biotech’s first therapeutic candidate, CEQ508 for the treatment of Familial Adenomatous Polyposis (FAP), is expected to enter into clinical trials in second half of 2010. The clinical trial will be conducted in FAP patients at Massachusetts General Hospital. For more information: