New Therapy Targeting microRNA-214 Blocks Ulcerative Colitis Disease Progression

New Therapy Targeting microRNA-214 Blocks Ulcerative Colitis Disease Progression

Scientists at the UCLA Jonsson Comprehensive Cancer Center developed a new therapy that blocks progression of ulcerative colitis and colon cancer in mice. Together with Crohn’s disease, ulcerative colitis is the most common type of Inflammatory Bowel Disease (IBD), a disease characterized by inflammation in the digestive tract — including the mouth, esophagus, stomach, small intestine, large intestine (colon), rectum, and anus and estimated to affect 1.4 million Americans. Colorectal cancer is a major cancer-leading cause of death in the United States, with the American Cancer Society estimating 93,090 new cases of colon cancer and 39,610 new cases of rectal cancer in the United States in 2015 alone.

The newly developed therapy uses a chemical drug that targets a specific small non-coding RNA molecule — microRNA-214 — which in high levels were previously found to occur in patients with ulcerative colitis. The study was performed over two years, where scientists Dr. Dimitrios Iliopoulos and his colleague Dr. Christos Polytarchou analyzed 401 colon tissue samples from patients in the United States and Europe with ulcerative colitis, Crohn’s disease, irritable bowel syndrome, sporadic colorectal cancer, and colitis-associated colon cancer compared them to those of healthy controls.

The team of scientists developed a “systems approach” where they combined sophisticated algorithms with high-tech robotics to screen new drugs targeting potential new key genes in a fast and accurate manner. This approach led researchers to identify a new drug that targets and inhibits microRNA-214 as a potential new treatment for ulcerative colitis and colon cancer.

Dr. Dimitrios Iliopoulos, an associate professor of digestive diseases noted, “The first steps of the drug discovery process usually take five to six years and by using our novel approach we expedited the drug discovery process only in two years. We evaluated this drug in mice with ulcerative colitis and colon tumors and found that in both cases it was highly effective to suppress these diseases.”

The team is performing additional studies with the newly identified microRNA-214 inhibitor to apply for the Food and Drug Administration (FDA)’Investigational New Drug Application. Additionally, authors hope to begin a Phase I Clinical Trial with ulcerative colitis patients next year.