Researchers are one step closer to developing personalized microbial therapeutic interventions for inflammatory bowel disease (IBD) thanks to a novel study co-sponsored by Cincinnati Children’s and two other pediatric institutions.

The study is exploring the potential benefits of the prebiotic 2'-fucosyllactose (2’FL) in restoring a healthy microbiome in children and young adults with IBD. It is the first National Institutes of Health-sponsored, randomized controlled trial to study 2’FL.

Phase one concluded in 2022. Study participants were young adults 18-25 with Crohn’s disease or ulcerative colitis who were doing well on Remicade (infliximab) or Humira (adalimumab). Two of the three doses tested met the required endpoints for safety, tolerability and effectiveness. In 2023, researchers at Cincinnati Children’s, Nationwide Children’s and Connecticut Children’s launched the study’s next phase, which is testing the two doses in children ages 11-17.

2’FL is an oligosaccharide that is present in breast milk. Synthetic 2’FL is available in powdered form and can be mixed into a beverage. Patients assigned to one of the 2’FL groups continue to follow their established treatment plan and take the supplement for one month (others take a placebo). Three months after their last dose, they return to the clinic for testing to see if their biome shows beneficial changes.

The Need for New IBD Treatment Strategies

IBD treatment has hit a plateau in recent years, says Lee (Ted) Denson, MD, principal investigator of the 2’FL study and director of the Schubert-Martin IBD Center at Cincinnati Children’s.

“There are more medication options than ever before, but a sizable number of patients don’t go into remission or they continue to have frequent flares,” Denson explains. “This study gets us closer to finding combinations of medication and nutritional supplements that will work better than medications alone—an approach patients and families are certainly eager to try.”

Scientists have known for some time that bacteria in the intestine drive gut inflammation and clinical relapses in patients with IBD. Drugs used to treat these conditions do not directly affect the microbiome. Prebiotics hold promise for restoring healthy gut microbiota in IBD and preventing clinical relapse. Denson and his colleagues theorize that 2’-FL supplementation will increase anti-inflammatory metabolites, including fecal Bifidobacterium and butyrate.

Probiotics and Prebiotics

Prebiotics are not used in the clinic setting, but many gastroenterologists prescribe probiotics containing Bifidobacterium for patients with ulcerative colitis. When the probiotics work, they replace harmful bacteria. But for about half of patients, the probiotics don’t remain in the digestive tract long enough to help, Denson says. Other institutions are working to develop more effective probiotics.

“Every patient is different, so we need to develop personalized treatment strategies for people with IBD,” he adds. “Different combinations of diet, medication and supplementation would help more people achieve intestinal healing and experience a better quality of life.”

To learn more about the 2’FL study, contact Lee.Denson@cchmc.org.