Crohn’s Research Focuses on Prevention

Research looking at what causes Crohn’s disease

biology research

Most research into Crohn’s disease is focused on treating rather than preventing its symptoms. But the work of Christine McDonald, PhD, a researcher at Cleveland Clinic’s Lerner Research Institute (LRI), is an exception.

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Crohn’s disease is a chronic inflammatory bowel disorder that affects 700,000 individuals in the United States. Inflammation, swelling or irritation may occur in any part of the digestive tract, causing abdominal pain and diarrhea. There is no cure; treatment includes medicines to reduce inflammation and relieve painful recurrent effects.

Scientists aren’t sure what causes Crohn’s disease but believe that it may result from an abnormal reaction by the body’s immune system. Dr. McDonald’s preliminary research has uncovered a way to address a fundamental mechanism causing the disease.

A different approach to Crohn’s

Scientists have known that a gene called NOD2 protects against Crohn’s disease. Dr. McDonald discovered that the NOD2 activity is controlled by an enzyme that can be blocked by an existing cancer drug, causing the protective activity of NOD2 to increase.

She is investigating the hypothesis that the effects of this drug will protect people from developing Crohn’s disease or will keep Crohn’s patients from suffering flare-ups of disease symptoms.

Because the drug already has been deemed safe in humans, the potential translation of Dr. McDonald’s laboratory studies into a pre-clinical trial can be greatly accelerated. Testing now is in a translational phase, focusing on the drug’s effect on cells from patients with Crohn’s disease.

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“Research moves slowly, and there’s a lot that we don’t yet understand,” Dr. McDonald says. “If we hadn’t received funding, we would be at least a year behind. That doesn’t sound like a lot, but it affects momentum, as well as the availability of scientists and postdoctoral researchers for our specific study.”

Competing for research funding

Dr. McDonald and her team recently were awarded a three-year, $750,000 grant from the U.S. Department of Defense to continue their research.

Federal budget reductions have forced research funding to become increasingly competitive, requiring extensive preliminary data and a proven track record from applicants. Less than the top 10 percent of applications are funded, Dr. McDonald says.

“Unfortunately, that often puts investigators into a ‘catch-22’ situation where, in order to get funding, you need significant preliminary data that you can’t afford to generate until you get funding,” she says.

Philanthropy helps fill the gap

The LRI Chairman’s Innovation Fund helps to fill this gap and provide critical resources to launch innovative projects, refine scientific approaches and recruit top-notch personnel.

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The Chairman’s Innovation Fund puts individual donations made to LRI to work collectively, awarding two research grants each year of $50,000 for the most promising projects submitted. The fund promotes early-stage research projects and opens channels to more significant funding as the concepts develop further.

Dr. McDonald received the Chairman’s Innovation Award in June 2011 and credits the funding as instrumental in receiving the DOD grant.

“Without the support of the LRI Chairman’s Innovation Award, my team would have had to delay working on this project until we had sufficient funds,” she says. “I can’t thank the donors to the Chairman’s Innovative Research Fund enough. It’s wonderful to see their enthusiasm and support for research.”

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