Using a new gene-editing technology, researchers at Shriners Hospitals for Children St. Louis and Washington University School of Medicine have re-wired living cells to produce their own targeted arthritis-fighting vaccine. These SMART (stem cells modified for autonomous regenerative therapy) cells could offer more targeted treatment options for children and adults with inflammatory and chronic conditions.
SMART cells can sense arthritis inflammation in a particular joint and make their own natural antidote to combat it, as needed. The research will be published Stem Cell Reports.
"For children with juvenile arthritis, this breakthrough could have significant benefits as many of the current treatment options cause unwanted side effects and may not be appropriate for children," Farshid Guilak, Ph.D., research senior author, director of research for the St. Louis Shriners Hospital and professor of orthopaedic surgery at Washington University School of Medicine said. "Our goal is to deliver a therapy that is self-regulating and targeted specifically to inflammation in a particular joint, as opposed to current drug therapies that impact the immune response throughout the entire body."
To date, SMART cells have only been engineered in a laboratory using cells from mice. Human clinical trials are a few years away.
Shriners Hospitals for Children is an international health care system providing pediatric specialty care, regardless of the families' ability to pay. As part of its mission to conduct research that improves the quality of care to children, Shriners Hospitals opened the Pediatric Musculoskeletal Research Center in St. Louis in 2016.