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Lupus: Systemic Lupus Erythematosus (SLE)

A Novel Approach: Stem Cells

A stem cell is unique in that it is a nonspecific cell type and has the potential to develop into many different types of specialized cells. These cells can divide and produce another stem cell to replenish itself or grow into a specialized cell, such as a nerve cell, brain cell, or a B cell.

Stem cell transplantation has the potential to revolutionize the treatment of several types of diseases. In this procedure, stem cells are taken from a person, grown in the laboratory into specialized cells, and then transplanted back into the individuals to replace diseased cells. To treat lupus, one approach is to take blood stem cells from a person with lupus and grow them in the laboratory into healthy new B and T cells that do not attack self-tissues. The next step is to replace the autoimmune B and T cells in an individual with the individual’s own new, healthy B and T cells.

This general approach is called autologous hematopoietic stem cell transplantation. The word "autologous" refers to the fact that the transplanted blood cells are derived from the person’s own stem cells; "hematopoietic" refers to the fact that the type of stem cell used is the precursor of blood cells like B and T cells. As of 2011, approximately 200 stem cell transplantations for the treatment of lupus have taken place.56

Data regarding the safety and efficacy of autologous stem cell transplantation is not yet plentiful, but some studies suggest that this treatment approach may be promising.

For example, in one small clinical study conducted in China, the disease status of almost 65% of patients did not get any worse over seven years.57 A comprehensive review of several studies that investigated autologous hematopoietic stem cell transplantation revealed that, in total, 81% of those that survived at least three years beyond the procedure showed some positive response to treatment.58 However, it is important to note that this analysis also found that an average of 11% of people who participated in these types of studies ultimately died because of transplant-related causes.

Stem cell transplantation is currently reserved for individuals with very severe disease who have not responded to conventional lupus treatments. In this population specifically, a remarkable 50% probability of 5-year disease free survival was achieved in the two largest studies to date exploring stem cell transplantation as a therapeutic option for lupus.56

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