Battling C. difficile
C. difficile strikes 24,000 children in the United States each year, and many more around the world. The number of U.S. cases is growing, prompting the federal Centers for Disease Control and Prevention to classify C. difficile as an immediate public health threat that requires aggressive action. The infection causes severe diarrhea and is especially dangerous to children with health conditions such as cancer, bowel diseases, and cystic fibrosis.
C. difficile is typically treated with antibiotics, but children who contract the infection often suffer repeated bouts. For some, it can become incurable, requiring the removal of the large intestine to prevent death.
Moonah, however, aims to protect children from becoming so terribly sick. He would do that by genetically engineering a single-celled amoeba often found in the gut called “Entamoeba.” Entamoeba is a parasite, but Moonah aims to use gene editing to turn it into a powerful ally against C. difficile.
Moonah plans to introduce a harmless form of Entamoeba into the intestine in order to directly deliver specific antibodies to inhibit damaging toxins produced by C. difficile. If his innovation works, this would be the first time an amoeba or other protozoan had been genetically engineered to deliver a treatment this way.
Ultimately, Moonah aims to turn his “protozoan technology and drug-delivery system” into a platform used routinely to deliver a wide range of drugs to battle gut problems in young children. The strategy could offer many benefits, he says, including lessening antibiotic use amid the growing problem of antibiotic resistance.
“If successful, there will be many potential applications to treat other intestinal diseases,” Moonah said. “The burden of C. difficile infection is a severe problem, and I am excited for the opportunity to help find new solutions that will lead to healthy children.”
About the Hartwell Award
Every year The Hartwell Foundation announces its Top Ten Centers of Biomedical Research in the United States, inviting each center to nominate individuals for a Hartwell Individual Biomedical Research Award. The Hartwell Foundation selected UVA as a center of excellence in a qualification process that considered the shared values the institution has with the Foundation relating to children’s health, while also considering the presence of the medical school, biomedical engineering, and the quality and scope of ongoing research.
All nominees for an award must submit a detailed research proposal to the foundation, undergo a personal interview, and make a presentation in defense of their early-stage, innovative, and cutting-edge applied biomedical research. The foundation seeks to support research that addresses a specific and compelling unmet need to improve health outcomes for children.
Moonah was one of only 10 scientists nationwide selected this year to receive a coveted Individual Biomedical Research Award.
“The Hartwell Foundation seeks to inspire innovation and achievement by offering individual researchers an opportunity to realize their professional goals,” said Fred Dombrose, president of the Memphis-based foundation. “Our approach is to be unique, selective, thorough, and accountable. We provide an opportunity for those we support to make a difference and to realize their hopes and dreams.”
Source: University of Virginia