Stem cell transplants are being investigated as a possible way to treating several autoimmune diseases, including scleroderma.

In scleroderma, the immune system mistakenly attacks healthy connective tissue. These attacks trigger damaging responses and lead to the buildup of collagen that causes hardened scar-like tissue to form.

There is currently no cure for scleroderma, but stem cell therapy may have the potential to alter or even “reset” the immune system and treat the disease.

What are stem cells?

During development, the cells in the body “differentiate,” or become specialized to be able to carry out a set and defined function. Once specialized, a cell generally cannot change into another type of cell.

But our stem cells, unlike the majority of the cells in our body, have the potential to develop into a range of different types of cells. These cells are able to maintain and repair tissues and organs. They can self-renew to produce more stem cells, and develop into the specialized cells of the tissues in which they are found. For example, they can replenish blood cells or repair the worn lining of cells of the small intestine. This specialization only occurs in stem cells under very specific circumstances, and in some organs — like the pancreas and heart — it is very difficult to trigger this change.

Types of stem cells

There are different types of stem cells with the potential to become different types of specialized cells, as stem cells have specific properties based on the tissue from which they are harvested. Stem cells are broadly split into two types: embryonic stem cells and adult stem cells.

Embryonic stem cells have the potential to develop into any cell type. They are obtained from a human egg cell, donated with consent, that is fertilized and cultured in the laboratory. The use of these types of cells is highly regulated.

Adult stem cells can be harvested from many tissues, but they are restricted in the type of cell they can become. There are many types, including:

  • Hematopoietic stem cells are related to blood and immune cells
  • Mesenchymal stem cells, which produce bone, muscle, and connective tissue, as well as regulating the immune response
  • Neural stem cells that can produce certain types of nerve cells
  • Epithelial stem cells that, for example, renew the walls of the small intestine

How could stem cells help in scleroderma?

In scleroderma, it is hoped that a stem cell transplant  might one day be able to modify or reset the immune response, potentially reducing or preventing disease progression. It is even possible that stem cells could be used at a future date to repair damage caused by scleroderma in tissues.

Stem cells in clinical trials for scleroderma

The two main targets being investigated for scleroderma so far are hematopoietic and mesenchymal stem cells. These have been demonstrated to be able to alter the immune response, and even potentially rebuild the immune system.

Hematopoietic stem cells have been investigated in several clinical trials. Most recently, results from a Phase 2/3 clinical trial (NCT00114530), called SCOT, in people with severe systemic scleroderma suggest that a hematopoietic stem cell transplant could one day be of significant clinical benefit.

Mesenchymal stem cells are still in the early stages of research, as no large-scale controlled trials have been carried out. However, early case studies suggest they could have a benefit and may have fewer side effects than hematopoietic stem cells. Further studies, and potentially clinical trials, are planned.

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