Using a new gene editing technology, researchers at Shriners Hospitals for Children-St. The Louis and Washington University School of Medicine has reconnected living cells to produce a targeted vaccine against arthritis. These stem cells modified for autonomous regenerative therapy (SMART cells) could offer more targeted treatment options for children and adults with inflammatory and chronic diseases.
Unlike current arthritis medications, which are administered continuously and at high doses, these SMART cells can detect inflammation of arthritis in a particular joint and make their own natural antidote to combat it, if needed.
Lead author of a study highlighting the work, published in Stem Cell Reports, says that children with juvenile arthritis could benefit significantly because many of today's treatment options cause unwanted side effects and may not be suitable to children. "Our goal is to offer self-regulating therapy specifically targeting inflammation in a given joint as opposed to current drug therapies that impact the immune response throughout the body," says Farshid Guilak, Ph.D., director St. Louis Shriners Hospital and Professor of Orthopedic Surgery at the University of Washington School of Medicine.
To date, SMART cells have only been designed in a laboratory using cells from mice. While human clinical trials are a few years away, the SMART cell method could also revolutionize treatments for many other conditions.
"The hope is that we can grow live bones, cartilage and whole joints that are resistant to arthritis and possibly other inflammatory diseases," says Guilak. "It could help adults and children with juvenile, rheumatoid, and other forms of arthritis, as well as other orthopedic conditions."
Last year, a Shriners Hospitals research team, led by Guilak, created a replacement for the hip joint of living tissue in patients receiving synthetic replacement joints that eventually wear out. This advancement is key for children who need to replace the joints because a living joint could grow with the child. SMART cell technology is also combined with this discovery to develop a living joint with these same disease-fighting properties.