Adult stem cells are able to restore self-tolerance, provide modified immune response (immunomodulation) and neuroprotection, and promote regeneration in patients with autoimmune diseases such as scleroderma.

Hematopoietic stem cell (HSC) transplants are being studied in two international trials, SCOT and ASTIS, to determine if stem cell therapy can reset the immune systems of scleroderma patients.

The use of MSCs as immunomodulating agents is also being explored. Preliminary results suggest that people with systemic scleroderma’s bone-derived MSCs exhibit effective in-vitro (laboratory) effects on lymphocytes.

Although stem cell therapy is not yet ready for all scleroderma patients, it has been proven effective in some situations when all other treatments failed. Scientists hope large scale studies will improve the approach.

MSCs (mesenchymal stem cells)

Studies in MSCs suggest that they act without causing any immune reaction, even when coming from a donor. As a result, MSCs can potentially “reset” and reboot the immune system. A case report of two patients with debilitating scleroderma revealed that they both benefited from combined immunotherapeutic techniques that included plasma exchange, rituximab, and stem cell therapy using MSCs. Both patients reported less pain, more mobility, less fatigue, improved breathing, and better overall quality of life.

The MSCs may have worked through immune modifications that balanced the perturbed cytokine and growth factor in scleroderma, adjusted immune cellular balance, and reduced ongoing fibrosis. In short, they changed the disease course.

HSCs (hematopoietic stem cells)

An HSC transplant is different from all other stem cell treatments because its primary goal is to replace and reset the entire immune system. All immune cells are regenerated after the “old” immune system is destroyed through radical immunosuppression via chemotherapy and other techniques.

HSCs can become neuronal cells that show development and protection effects. They can be collected directly from the bone marrow and then transplanted back into the patient’s body (autologous HSC transplant), or they can be transplanted into a different person.

Studies from Europe and the U.S. have shown dramatic and durable improvements in skin fibrosis and quality of life of people with scleroderma via autologous HSC transplant. The ASSIST and ASTIS studies both showed clinical benefits after transplantation. The SCOT study is still following participants.

NSCs (neural stem cells)

NSCs can provide a source to structurally repair the central nervous system.

Experiments have been done in animal models with immune diseases, but not scleroderma. The NSCs were injected either intrathecally (into the spine) or via a ventrical to place the cells into the brain’s cerebrospinal fluid compartment (bypassing the blood-brain barrier).

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