In February, we reported on Dr. Farshid Guilak’scurrent Arthritis Foundation-funded trailblazing project, “Engineering New Biologic Therapies for Arthritis.”
Dr. Guilak’s work is published in the journalStem Cell Reports. The paper, entitled “Genome engineering of stem cells for autonomously regulated, closed-loop delivery of biologic drugs,” describes the research team’s method of engineering (reprogramming) stem cells to become “smart stem cells” that will sense inflammation and deliver biologic drugs where they are most needed. The smart stem cells-biologic drug combination is then injected into an arthritic joint.
“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, but only when it is needed,” Dr. Guilak explained. “To do this, we needed to create a ‘smart’ cell.”
Once the inflamed cells are identified, the smart stem cells incorporate themselves, not only to repair damaged cartilage, but also to produce and deliver specific therapeutic drugs to stop the inflammation. The smart stem cells are programmed to be active only when and where inflammation is present.
He explained that this application is currently aimed at inflammatory diseases likerheumatoid arthritis(RA) and that the advantage of using smart stem cells is that they can be individually designed to deliver just the right amount of therapy, instead of a constant, long-term exposure to very strong anti-inflammatory drugs. Dr. Guilak said that the smart stem cells will stop the inflammation and repair damaged cells at the same time.
研究小组还发现，在小鼠模型中，由工程设计的智能干细胞修复的软骨受到保护，不受炎症的影响。研究小组观察到，新生成的软骨细胞改变了炎症途径，并产生了一种保护药物。
For his next steps with this technology, Dr. Guilak and his team will test the engineered smart cells in larger animals and then, if the results continue to be positive, move to clinical trials in humans. The team’s goal is to have the engineered cells or the newly grown synthetic cartilage from stem cells to respond to inflammation by releasing a biologic drug that would protect the synthetic cartilage cells and the natural cartilage cells in specific joints.
Dr. Guilak and his team feel that this research has tremendous applications that can be adapted for therapy for other inflammatory diseases, such asjuvenile arthritis(JA) orpsoriatic arthritis(PsA). “The inflammatory processes are different for these diseases than RA,” he explained. “So, we need to adjust the programming.”
According to Dr. Jonathan Brunger, research team member and first author of the paper, “The ability to build living tissues from smart stem cells that precisely respond to their environment opens an exciting area of investigation for regenerative medicine.”
Dr. Guilak is a professor at Washington University Department of Orthopedic Surgery and is Director of Research for Shriners Hospitals for Children in St. Louis. He is also the co-director of the Washington University Center of Regenerative Medicine and has appointments in the Departments of Developmental Biology and Biomedical Engineering. He has received funding from the Arthritis Foundation for three separate projects over the past 10 years.
Can I get in on this trial? thanks
Would this vaccine also work for osteoarthritis, and approximately how long till it would be available?
如果有什么临床试验正在进行，我愿意做志愿者。我已经退休了，身体很好(除了RA -哈哈哈)。我在2017年2月被诊断出患有严重的风湿性关节炎。17年2月23日我刚开始用Plaquenil;17年5月25日开始在奥伦西亚工作。将于6月22日进行第三次注射(之后每月注射一次)，我对它的耐受性很好。
Such encouraging news! That would be a miracle!
PLEASE EXTEND THIS RESEARCH TO OSTEOARTHRITIS!! I am witnessing first hand the debilitation, excruciating pain and hopelessness it is causing in both my Mother and older sister. Especially osteoarthritis of the hands and spine. Unfortunately, I am also now beginning to experience what my Mother and Sister have been dealing with for many years. I have been donating to the foundation for almost 30 years and it saddens me that no significant improvements or progress in treatment for Osteoarthritis have been found.