Scleroderma is an autoimmune disease where the patient’s own immune system mistakenly attacks healthy tissues, leading to hardened scar-like tissues. In the severe form of this disease, known as systemic scleroderma, scar tissue can also build in internal organs that include the heart, kidneys, lungs, and gastrointestinal tract.
There is currently no cure for scleroderma, but stem cell therapy may be an effective future approach to treating this disease. Mesenchymal stem cell transplants are one type of this therapy under investigation.
How mesenchymal stem cell therapy works
Most cells in the body are differentiated, meaning that they have developed to fulfill a fixed role, and generally cannot change to fill any other roles. But stem cells have the potential to develop into a range of other cells.
Mesenchymal stem cells can regulate the way the immune system works. They can reduce the growth of immune cells, and they may be able to “reset” the damaging immune response seen in scleroderma patients.
Mesenchymal stem cells are commonly harvested from bone marrow or the umbilical cord, and then cultured in the laboratory. They usually have a lower chance of rejection when they are transplanted into a patient compared to hematopoietic stem cells because of the way they alter the immune response, potentially making them a safer treatment option.
Mesenchymal stem cells may also be referred to as mesenchymal stromal cells.
Studies of mesenchymal stem cells in scleroderma
Mesenchymal stem cell transplants have not been invested as thoroughly as hematopoietic stem cells. However, although more work is needed, a few case studies and small clinical trials have been conducted, and suggest they may be beneficial to patients with scleroderma.
One case study assessed the effect of mesenchymal stem cells obtained from a matched donor (allogeneic) in two patients with progressive and treatment-resistant systemic scleroderma. Before the transplant, both patients underwent plasmapheresis, which filters the immune cells and proteins out of the blood, and a single dose of rituximab, which suppresses the immune response.
Results, published in the journal Stem Cell Investigation in 2016, showed that following the stem cell transplant, the patients’ symptoms improved; they had reduced lung symptoms such as shortness of breath, lesser joint pain, and greater joint flexibility. Their overall mobility and quality of life also improved. Both patients opted to receive a second stem cell transplant after disease progression at 12 and 16 months, respectively.
An open-label Phase 1/2 clinical trial (NCT00962923) is assessing the safety and efficacy of allogeneic mesenchymal stem cell transplants in 20 patients with systemic scleroderma. The trial is taking place at a single site in China, but it is not clear if it still underway.
The stem cells were obtained from umbilical cords and cultured in the laboratory. Treated patients underwent three rounds of plasmapheresis before receiving a single dose of the cells.
Results from 14 patients (of 20 planned for enrollment) were published in 2017 in the journal Arthritis Research & Therapy and showed that — at one year of follow-up — their skin symptoms had significantly improved, measured by the modified Rodnan skin score. Three patients with interstitial lung disease (ILD) due to scleroderma also showed significantly improved lung function. No serious adverse events were reported.
An open-label Phase 1/2 trial (NCT02213705) in France is recruiting 20 cyclophosphamide-resistant systemic scleroderma patients to assess the safety and efficacy of allogeneic mesenchymal stem cell transplant. The trial is expected to be completed in June 2019.
Mesenchymal stem cell transplant for digital ulcers
A randomized, double-blind, controlled Phase 1/2 clinical trial (NCT03211793), called MANUS, aims to assess the safety and efficacy of local mesenchymal stem cell injections as a treatment for digital ulcers in systemic scleroderma patients. Digital ulcers, sores on fingers and toes that do not heal, are a common symptom of scleroderma, as the disease can cause restricted blood flow to the extremities.
This trial is expected to start enrolling up to 20 participants at a single site in the Netherlands in November 2018. It is expected to conclude in November 2020.
Another trial (NCT02975960) investigating a local injection of mesenchymal stem cells to treat digital ulcers in systemic scleroderma ended in January 2018. This trial enrolled up to seven patients at a single site in South Korea, given a single injection of mesenchymal stem cells taken from their own adipose tissue, or body fat. Results have not yet been published.
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