Systemic lupus erythematosus is an autoimmune disease that affects primarily young women of color during their reproductive years.
Only one drug has been approved for treating lupus in the last fifty years. Current therapies have limited efficacy and significant toxicity especially regarding reproductive health. Mesenchymal stromal cells (MSCs) can be derived from a number of sources including bone marrow, umbilical cords or adipose tissue. Collaborators in China have treated over 100 patients with lupus refractory to standard therapy with MSCs from umbilical cords and report a 75% response rate with minimal toxicity.
In collaboration with these Chinese investigators, we were able to transfer the technology needed to produce the cells to the MUSC Center for Cellular Therapy (CCT). We performed pre-clinical testing of the cells in cell culture and in animal models of lupus and found similar significant immune effects and amelioration in disease in mice as reported by our Chinese colleagues.
Based on these findings, we performed a Phase I trial of umbilical cord derived MSCs in 6 lupus therapy. From these studies, we were able to obtain funding from the Lupus Foundation and the NIH to perform a double blind placebo controlled multicenter trial of MSCs for the treatment of 81 patients with refractory lupus. Six other centers are participating including Emory, UNC, Rochester, Northwestern, UCSD and Cedars Sinai in LA. All the cells are produced in the MUSC CCT facility.
Having a facility like the CCT is critical for this project. With this funding, a study of over eight million dollars will be performed over the next five years. Dr. Wang, Ms. Duke and Ms. Cloud of the CCT were critical to developing the techniques for cell derivation and for getting approval from the FDA to perform the trial. The funding of the trial would also not have occurred without the proven excellence and certification of the facility.
Enthusiasm for such cellular therapies is off the wall as we have had over 100 inquiries from patients wanting to participate in the trial. Only through such trials can the efficacy of a new therapy be proven. The immune studies will provide us with critical clues as to the underlying factors that contribute to lupus and perhaps markers that will define which patients will respond to this treatment and which may not.
Gary Gilkeson, M.D.