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A novel clinical trial at Children’s Hospital Los Angeles is investigating whether butyrate—a short-chain fatty acid typically produced by gut bacteria—can be a potential therapy for children with ulcerative colitis.
The pilot study, which plans to enroll 20 pediatric patients, is funded by a grant from the Crohn’s & Colitis Foundation Litwin IBD Pioneers Initiative, which supports innovative clinical and translational research in inflammatory bowel disease (IBD). The trial is the first to evaluate butyrate in children with ulcerative colitis, a form of IBD—and the first to focus on Hispanic youth with the disorder.
“Many Hispanic children with ulcerative colitis develop more severe disease, leading to hospitalizations, blood transfusions, infections and even surgery,” says Sonia Michail, MD, FAAP, CPE, AGAF, Principal Investigator for the study and Director of Research for the Inflammatory Bowel Disease Program at Children’s Hospital Los Angeles. “Our hope is that butyrate could be a therapy that helps with their disease control.”
The idea for the study grew out of Dr. Michail’s recent research into the gut microbiomes of children with ulcerative colitis.
In that study—published last year in Physiological Reports—she and her team examined stool samples from 22 Hispanic and 31 non-Hispanic children with the disorder. The team found that the Hispanic children were strikingly deficient in butyrate.
“They were really depleted,” she says. “There wasn’t much butyrate at all that was being produced by the bacteria in their intestines. That was very different from the non-Hispanic children, who made less butyrate than healthy children but were not nearly as deficient.”
That prompted Dr. Michail to take a closer look at butyrate as a potential therapy. The short-chain fatty acid is known to play a role in protecting the health of colonocytes, the cells that line the large intestine.
“The idea is to give them back what they’re deficient in,” she explains. “If the bacteria in your intestine are not producing much butyrate, then you’re not getting that protective effect.”
In the 1990s, butyrate supplementation was studied in adults with IBD with mixed results. Some patients showed significant improvement, while others experienced no effect at all. However, those studies were concentrated in Caucasian patients, and they didn’t measure patients’ prior levels of butyrate.
“Perhaps the people who benefited had been deficient, and the ones who didn’t respond already made enough butyrate on their own,” Dr. Michail says. “These studies were also not done in Hispanic patients, and that is the group we have found to be most lacking in this nutrient.”
The randomized controlled trial, which is now actively enrolling patients, is open to children of all ethnicities. But the team expects to enroll a high number of Hispanic children, who make up a large percentage of the hospital’s patient population.
Children in the study will receive 60 milliliters of sodium butyrate daily for three months. The dose, which is administered via an enema, works directly on the intestinal tissue. A small number of children will be randomized to a placebo group.
In addition to measuring participants’ butyrate levels, the team will monitor disease activity in multiple ways—including biopsies, bloodwork and patient questionnaires—before, during and after treatment.
After the pilot study is complete, the researchers plan to conduct a larger study with an additional 50 children.
Dr. Michail adds that she is particularly excited by butyrate’s potential to come with minimal to no side effects—a very different picture from current ulcerative colitis treatments.
“The types of medications we currently use in these patients suppress the immune system and come with a very long list of side effects,” Dr. Michail notes. “Our hope is that butyrate can help control disease without causing those side effects. That would be a big step forward in improving care for these children.”