Introduction: What is Scleroderma?
Scleroderma is a rare autoimmune disease in which the body’s immune system mistakenly recognizes your connective tissue as a pathogen and attacks it. As a result, this leads to fibrosis, or the overproduction of collagen and scar-like tissue. Scleroderma comes in two different forms: localized scleroderma, which only affects the skin; and systemic sclerosis, which affects both the skin and internal organs. Of the two, systemic sclerosis is more fatal, since the formation of scar-like tissue in the internal organs can lead to organ dysfunction. To make matters worse, there is currently no cure for scleroderma, but there are treatments available to help alleviate the symptoms. Of these treatments, stem cell therapy is currently being explored as a potential treatment for managing scleroderma.
How can Stem Cells Help Treat Scleroderma?
Stem cell therapy involves transplanting stem cells—unique cells with the ability to differentiate into specialized cells—to a desired location in the patient’s body. By transplanting stem cells, researchers hope that it will modify or reset the immune response, potentially slowing or stopping the progression of scleroderma. Researchers also hope to someday utilize stem cell therapy as a form of regenerative medicine for scleroderma, repairing the damage caused by the scar tissue on the organs.
Example: The SCOT Clinical Trial
There are ongoing clinical trials regarding stem cell transplantation in scleroderma patients, with a primary focus on transplanting hematopoietic stem cells, which differentiate into all types of blood cells, and mesenchymal stem cells, which differentiate into cartilage, bone, and the fat found in the bone marrow. In a Phase 2/3 clinical trial named SCOT (Scleroderma: Cyclophosphamide or Transplantation), stem cell transplants have shown superior long-term benefits compared to the standard treatment for severe scleroderma. The study involved 75 patients with systemic sclerosis, 97% of whom had lung involvement, at 26 clinical sites. The patients were randomized and divided into two groups: one receiving myeloablative autologous hematopoietic stem cell transplants, involving high-dose chemotherapy and stem cell infusion; and the other receiving the standard treatment, monthly doses of the drug cyclophosphamide.
Results from the trial indicated that the stem cell transplant group experienced significant improvements, with 67% showing better global disease symptom assessments compared to 33% in the cyclophosphamide group. After 4.5 years, 79% of transplanted patients did not show disease progression, while only 47% in the cyclophosphamide group did. The overall survival rate was 86% in the transplant group compared to 51% in the cyclophosphamide group after 72 months. Although both treatments carried similar risks of infection and low blood-cell counts, the transplant group had a higher incidence of cancer, potentially linked to radiation therapy. Despite this, the researchers concluded that myeloablative autologous stem cell transplants led to significantly better clinical outcomes than the standard treatment, and the potential side effects should be weighed against the treatment benefits.
Conclusion: Future of Stem Cell Therapy in Scleroderma Treatments
In conclusion, the exploration of stem cell therapy as a potential treatment for severe scleroderma presents a promising avenue in medical research. Stem cell therapy, involving the transplantation of unique cells capable of differentiating into specialized cells, offers hope for modifying or resetting the immune response associated with scleroderma. Ongoing clinical trials, particularly the Phase 2 SCOT trial, have demonstrated that myeloablative autologous hematopoietic stem cell transplants can provide superior long-term benefits compared to standard treatments like cyclophosphamide. Notably, the transplant group exhibited significant improvements in symptoms typical of systemic sclerosis, a lower rate of disease progression, and a substantially higher overall survival rate. It is likely that in the near future, stem cell therapy will become an approved treatment—not just an experimental one—and thus will become more accessible for scleroderma patients.
Written By: Paul Tran
[Image] Chatterjee, S., & Majhail, N. (2018). Autologous Hematopoietic Cell Transplantation:
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