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.
Unlike current arthritis medications that are given continuously and at high doses, these 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 in the May 9 issue of the journal, 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,” said 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. “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 still a few years away. The SMART cell method could also revolutionize treatments for many other conditions.
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.
Last year, a Shriners Hospitals’ research team lead by Dr. Guilak created a living tissue hip joint replacement for patients currently receiving synthetic replacement joints that eventually wear out. This advancement is key for children who need replacement joints since a living joint could grow with the child. SMART cell technology is also being combined with this discovery to grow a living joint with these same disease-fighting properties.
“The hope is that we can grow living bone, cartilage and whole joints that are resistant to arthritis and eventually other inflammatory diseases too,” said Dr. Guilak. “This could help adults and children suffering from juvenile, rheumatoid and other forms of arthritis, as well as other orthopaedic conditions.”