Stem Cells Have Promise for SLE Treatment Across Ethnicities, Proof-of-concept Study Shows

A new proof-of-concept study supports the use of mesenchymal stem cells (MSCs) to treat systemic lupus erythematosus (SLE), showing that transplanting MSCs can control the disease in patients from different ethnicities.

The study, “Therapeutic potential of allogeneic mesenchymal stromal cells transplantation for lupus nephritis,” was published in Lupus.

MSCs are cells that are able to differentiate (transform) into certain other types of cells, including bone, muscle, and fat cells. These cells are thought to have potential as treatments for SLE because they can reduce bone loss and inflammation in mouse models.

However, there is limited data about how well MSC therapy works in humans. There have been a few studies with promising results, but these have come almost entirely from centers in China, meaning the patients are share the same ethnic background.

In this proof-of-concept study, MSC therapy was used for three patients in Spain. Two of the patients were male Spanish Caucasians; the third was a Bolivian female of indigenous descent. All patients had been diagnosed with SLE for more than a decade and, at the time of treatment, had flare-ups that were not being well-controlled with standard therapies.

MSCs were taken from the bone marrows of healthy donors, purified, expanded, and then infused into patients intravenously at a dose of 1.5 million cells per kilogram of body weight — an average of 90 million cells per patient.

The patients were followed for nine months. Researchers assessed markers of inflammation and immune activity, as well as markers of kidney function like the amount of protein in their urine. Kidneys are often targets of the body’s autoimmune attack in SLE, and damage to them can be a serious issue in this disease. The researchers also measured disease severity using the SLE Disease Activity Index 2000.

All three patients responded to treatment with MSCs. Two had complete responses, with decreased inflammatory markers, signs of better kidney function, and lower disease score that were sustained over the entire nine-month follow-up period. The third was only deemed a partial response, but was still an improvement, and dosages of other medications could be reduced by 50% to 90% for all the patients.

The researchers reported there were no adverse side effects or safety problems associated with the patients receiving the MSC therapy. There also was no evidence that the MSCs were being rejected by the body, which can be a concern for these types of therapies.

“Our results confirm the successful results of MSC treatments in SLE patients performed in different ethnic groups and locations,” the researchers concluded in their paper, also noting that the findings support the prompt implementation of a Phase 2 clinical trial testing the approach in a larger group of patients.

The trial (2017-000391-28), already approved by the Spanish Medicines Agency, will include 36 patients with lupus nephritis (a common kidney inflammation in SLE patients) and randomly assign them an infusion with MSCs or a placebo.

The trial’s main objective is to determine if more patients respond to MSCs. Secondary measures include the time to response, duration of response, safety, and reductions in use of corticosteroids and immunosuppressants.