Treating patients with drug-resistant progressive scleroderma with a combination of immunotherapy and umbilical cord-derived mesenchymal stem cells (MSCs) could improve their condition, according to a study reporting the case of two patients published in the journal Stem Cell Investigation.
According to the authors of the study “Mesenchymal stem cell therapy for refractory scleroderma: a report of 2 cases,” the results suggest that a large-scale study is necessary and could further prove the beneficial effect of this combination in the treatment of drug-resistant progressive scleroderma.
The team, led by Dr. Talar Margossian of The Lebanese University Faculty of Medicine in Beirut treated two drug-resistant progressive scleroderma patients, ages 28 and 30, with plasmapheresis, a technique where the blood plasma containing harmful antibodies for the body is removed by a machine and replaced by healthy plasma without such antibodies.
Patients were then given the immunosuppressive drug Rituxan (rituximab), which is an antibody against the CD20 protein found on the surface of B-cells of the immune system, destroying them. Finally, the patients received umbilical cord-derived MSCs injection.
Researchers reported that both patients showed an improvement for more than a year, in which their pain was resolved, they were more mobile, and experienced less fatigue and shortness of breath following mild exercise.
The team also assessed the two patients using the European Scleroderma Study Group (EsSG) activity index, which is an objective measure of disease activity. They found that the index was reduced following treatment, indicating an improvement in disease activity.
However, the patients’ symptoms recurred after a year, when they were given a second course of MSCs. This led to a good response where patients reported improved mobility, function, and quality of life.
The researchers concluded that a combination of immunosuppressive therapy and MSCs treatment is effective and well tolerated by scleroderma patients.
The team speculated that MSCs likely work through immune modulation in which they “balance the perturbed cytokine and growth factor soup in scleroderma,” and adjust the immune cellular balance, therefore changing the course of the disease. However, a possible placebo effect on these results cannot be discounted.
“Stem cell therapy is not ready for use on every patient with scleroderma,” the authors wrote. “But there are reasons to believe that it is possibly effective where there is indeed no hope to offer.”
They added: “Future large scale studies can bring more scientific evidence to further improve this approach and fulfill the unmet needs to treat advanced scleroderma patients.”