Innovative efforts at the University of Virginia School of Medicine to use a harmless amoeba to protect children from dangerous infections has won critical financial support from The Hartwell Foundation.
The Hartwell Foundation, which supports early-stage, cutting-edge biomedical research to benefit children of the United States, will provide UVA researcher Shannon Moonah, MD, $100,000 each year for three years. The funds will support his effort to engineer an amoeba – a single-celled organism – to deliver antibodies that can prevent and treat potentially deadly C. difficile infections in young children.
New options are needed for C. difficile – there is no vaccine to protect at-risk children, and treatments for severe disease are suboptimal. Cell-based therapies like amoeba hold the promise of safe and effective drug delivery systems. I am beyond grateful for the generous support of The Hartwell Foundation to help develop this novel approach."
Shannon Moonah, physician-scientist, UVA's Division of Infectious Diseases and International Health
"We are delighted to have this vitally important research honored by The Hartwell foundation," said Melur "Ram" Ramasubramanian, UVA vice president for research. "Shannon Moonah is a great example of the innovative and exciting biomedical researchers at UVA."
About 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 infections are typically treated with antibiotics, but children who contract the infections offer 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 deliver directly 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 to receive a coveted Individual Biomedical Research Award this year.
"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."
Moonah's collaborators on the project at UVA will include Carol Gilchrist, PhD; Debbie-Ann Shirley, MD; and Cirle Warren, MD.