Investigators at Washington University School of Medicine in St. Louis, Shriners Hospital for Children in St. Louis, Duke University, and Cytex Therapeutics Inc. have developed a technology using so called SMART cells (Stem cells Modified for Autonomous Regenerative Therapy) that will develop into cartilage cells and at the same time produce a biologic anti-inflammatory drug, which will protect joints from the damage that results from chronic inflammation. Using a gene-editing tool known as CRISPR, the investigators remove a specific gene that is involved with inflammation and replace it with a gene that will release a biologic drug that fights inflammation.
The leader of the team of investigator, Farshid Guilak stated, “our goal is to package the rewired stem cells as a vaccine for arthritis, which would deliver an anti-inflammatory drug to an arthritic joint predominantly when it is needed”. The use of the term “vaccine” is unclear as a vaccine is a “a substance used to stimulate the production of antibodies and provide immunity against one or several diseases prepared from the causative agent of the disease, its products, or a synthetic substitute treated to act as an antigen without inducing the disease”. Thus, despite the creativity of this new technology, it is unclear how it forms the basis for a vaccine. Nonetheless, it is exciting and could make a meaningful difference to the way physicians treat arthritis or the way arthritis is manifest among patients.
Most current drugs that treat arthritis are aimed at an inflammation-promoting molecule known as tumor necrosis factor alpha (TNF-alpha). However, in order to combat TNF-alpha, drugs must be given systemically rather than targeted to joints. Consequently, the drugs may interfere with the immune system throughout the body and can make patients susceptible to side effects such as infection. Guilak states, “We want to use our gene editing technology as a way to deliver targeted therapy in response to localized inflammation in the joint as opposed to current drug therapies that can interfere with the inflammatory response throughout the entire body”.
Research such as this will hopefully one day make a meaningful difference for the lives of patients who are afflicted with arthritis.
Brunger JM, Zutshi A, Willard VP, Gersbach CA, Guilak F. Genome engineering of stem cells for autonomously regulated, closed-loop delivery of biologic drugs. Stem Cell Reports. Published online April 27, 2017.