A Game-Changer for Lupus?

Mesenchymal Stem Cells as Targeted Therapy for Autoimmune Disease

by Kimberly McGhee

The first clinical trial in the U.S. using allogeneic mesenchymal stem cells (MSCs) to treat human disease is being led by MUSC Health rheumatologists Gary S. Gilkeson, M.D., and Diane L. Kamen, M.D., MSCR, who are studying their safety and efficacy in patients with systemic lupus erythematosus (SLE), a chronic, relapsing, multi-system autoimmune disease. The phase 2 MSCs in SLE trial (MsciSLE; NCT02633163) will randomize patients with treatment-refractory SLE to standard of care plus a single IV infusion of low-dose MSCs (1 million MSCs per kilogram), high-dose MSCs (5 million MSCs per kilogram), or placebo. It will open first at MUSC Health but will ultimately be joined by five other sites—Emory University, the University of North Carolina at Chapel Hill, the University of Rochester, Northwestern University, and Cedars Sinai. The Center for Cellular Therapy at MUSC, which houses a cGMP Class 6–compliant clean room suite, will supply the MSCs, harvested from donated umbilical cords, for infusion at all study sites.

In autoimmune diseases such as SLE, the immune system, meant to protect the body against foreign invaders, loses its ability to distinguish between self and other and begins to attack its own tissue. Mesenchymal stem cells (MSCs), which secrete immunomodulatory factors that help restore immune balance, could hold promise for treating these diseases.1 Because MSCs do not express major histocompatibility complex 2 or costimulatory molecules, they are also “immunologically privileged” and less likely to be rejected after transplant.1

Most existing medications control SLE symptoms by suppressing the global immune system but at the cost of an increased risk of infection. In contrast, MSCs preferentially migrate to sites of inflammation, offering a more targeted therapy for autoimmune disease with fewer of the systemic adverse effects.

Promising results from uncontrolled trials of MSCs in autoimmune diseases in China have created excitement over this novel approach to SLE and fueled Gilkeson’s interest in trying to replicate these findings in a placebo-controlled trial (MsciSLE).

Lingyun Sun, M.D., Ph.D., of the Affiliated Drum Tower Hospital of Nanjing University Medical School, a sister hospital of MUSC Health, reported four-year results of a clinical trial of MSC infusion in 87 patients with severe active SLE. Remission rates were 28 percent at 1 year (23/83), 31 percent at 2 years (12/39), 42 percent at 3 years (5/12), and 50 percent at 4 years (3/6).2

“These impressive data suggest MSC therapy could be a low-risk, effective treatment for refractory lupus,” said Gilkeson. “However, a number of lupus therapies showed early promise only to later fail to show efficacy. That it is why a controlled trial is urgently needed.”

Gilkeson is currently enrolling patients in a small phase 1 trial funded by the Lupus Foundation of America to establish the safety of MSC infusion in patients with SLE. These patients will be followed for six months to monitor for treatment-related adverse effects. If the MSC infusions prove safe, as is expected based on Chinese experience using the same dose, then the phase 2 trial will open at MUSC Health and, as funding allows, at the other approved sites. Results are expected in 2021. For more information on this or other lupus trials, contact study coordinator Carol Lambourne.


1 Cras A, et al. Arthritis Research & Therapy 2015;17:301.
2 Wang D., et al. 
Cell Transplantation 2013; 22: 2267–2277.