Mesenchymal Stromal Cell Research

Stromal cell therapy is a promising frontier in lupus research, especially for people whose lupus doesn’t respond to other treatments (called refractory lupus).

We were the first lupus organization in the U.S. to support stromal cell research. In partnership with the National Institute of Allergy and Infectious Diseases, we committed nearly $4 million over five years to co-fund a major study of mesenchymal (adult) stromal cells as a new treatment for moderate to severe lupus.

This study is the first step in determining whether stromal cell therapy is safe and effective for people with lupus who do not benefit from currently available treatments. The study will determine whether therapy with mesenchymal stromal cells (MSCs) can:

• lessen the debilitating long-term effects of lupus
• reduce the need for medications like steroids, which have harmful side effects
• stop damage to vital organs

In 2018, the Lupus Foundation of America (LFA) agreed to provide $3.8 million to support a Phase II clinical study of stromal cells as a potential new therapy for lupus. In the preceding Phase I clinical trial, solely funded by the LFA, investigators at the Medical University of South Carolina sought to determine if MSCs therapy was safe to use in people with lupus. Six women with refractory lupus were treated with MSCs derived from umbilical cord tissue. While some patients reported nausea, tingling and flushing, there were no serious adverse events in response to MSCs treatment. Overall, five of the six women achieved a clinically meaningful decrease in lupus disease activity scores as measured by the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI). This decline in SLEDAI was sustained for 52 weeks. Steroids were also reduced to or maintained at 10mg or less per day in five of the six patients. One patient withdrew at the eight-week visit to seek other treatment for refractory disease.

Based on these early promising results, the study has now transitioned to a Phase II trial, which will include up to 81 study participants. The COVID-19 pandemic had a major effect on trial operations as investigators had to shut down enrollment for close to six months. Still, as of June 2022, investigators have treated 50 of the 81 patients needed. Two new sites were added this year to the other seven sites, including Oklahoma Medical Research Foundation in Oklahoma City, OK and the Feinstein Institute in Manhasset, NY.

Watch the video to learn more.