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Study to Explore Blood Stem Cells as Therapy for Severe Lupus

By Cheryl Simon Silver

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Stem Cells

Over the next five years, a pilot study at the U.S. National Institutes of Health will test the use of stem cells from blood as a therapy for people with severe cases of lupus, a disease in which a person’s immune system attacks the body’s cells and tissues. People with lupus may experience inflammation, joint pain and, in some cases, damage to major organs.

The therapy involves collecting stem cells—cells produced by the bone marrow that develop into blood cells—from the patient, completely shutting down the patient’s immune system, and then giving the patient back his or her stem cells.

After the immune system is weakened and the stem cells are returned, the researchers will monitor the new immune system through blood work. “We want to know, ‘Can the patient build a normal immune system?’” says Gabor Illei, a rheumatologist at the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) in Bethesda, Maryland.

Participants in the study will receive a widely used chemotherapy drug called cyclophosphamide in combination with other medications. The reason for using several medications is to “achieve a more nearly complete deletion of the immune system without permanently harming the bone marrow,” says Illei.

The study was developed jointly by NIAMS and the National Cancer Institute, also in Bethesda, Maryland. Blood stem cell transplants are seen as potential treatments for maladies ranging from lymphoma to severe Crohn’s disease to multiple sclerosis.

The pilot study will involve fourteen individuals whose lupus is severe and resistant to conventional treatments. They will be monitored for five years to gauge how their immune systems respond to the treatment, and to establish if the blood stem cell treatment is safe in people with lupus.

For more information visit NIAMS.

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