Inhibition Drug Offers Specialized Treatment for Digestive Diseases

Caregivers can have trouble differentiating ulcerative colitis from Crohn's disease and inflammatory bowel disease.

UCLA scientists have fast-forwarded the drug discovery process for ulcerative colitis (UC) through a new systems approach.

The team, led by UCLA Jonsson Comprehensive Cancer Center member Dimitrios Iliopoulos, PhD, and his colleague Christos Polytarchou, PhD, found an experimental therapy treatment to suppress UC with a micro-RNA-214 inhibitor.

UC, which causes inflammation in the digestive tract and can lead to colon cancer, is sometimes difficult to diagnose. In many cases, physicians cannot differentiate UC from similar conditions, such as Crohn's disease and inflammatory bowel disease (IBD).

The scientists took 401 samples of patients with UC, Crohn's, colorectal cancer, and indeterminate IBD, and compared them with unaffected tissues. The team developed a systems approach with advanced computer technology and robotics to identify the most important genes to target for certain diseases, making an inhibitor drug easier to find.

"The first steps of the drug discovery process usually take 5 to 6 years and by using our novel approach we expedited the drug discovery process only in 2 years," said Iliopoulos, an associate professor of digestive diseases at UCLA.

This process led them to discover micro-RNA-214, which was present in high levels for patients with UC. In mice with UC and colon tumors, the micro-RNA inhibitor was highly effective in suppressing the disease. With this technology, specialized treatment options for UC patients may be available very soon.

Colorectal cancer is the third leading cause of cancer-deaths in the United States. The American Cancer Society estimates that 93,090 new cases will be diagnosed in the United States in 2015, and almost 50,000 people are expected to die from the disease this year.

Iliopoulos hopes to begin phase 1 clinical trials for UC patients within the next year, and to apply for a new drug application with the FDA.

This study was published online in Gastroenterology. It will appear in print October 2015.