Self Help: Stem cells rescue lupus patients

By rebuilding a patient’s immune system using his or her own stem cells, doctors can reverse the course of lupus in severely ill patients for whom medication no longer works, a new study shows.

In this autoimmune disease, white blood cells go awry and create antibodies that target the person’s cells and tissues. The tissue damage results in rashes, swollen joints, fever, and fatigue. Lupus can also turn deadly and attack vital organs, especially the kidneys, lungs, and nervous system.

Immune-suppressing drugs can alleviate symptoms, but they sometimes have debilitating side effects. In many patients, the drugs eventually stop working.

The experimental therapy uses stem cells to make new, healthy white blood cells to replace the tissue-targeting ones. Doctors first administer a drug that coaxes those cells out of a patient’s bone marrow into the bloodstream. The researchers isolate these stem cells from the patient’s blood.

The patients then receive drugs that wipe out the remaining defective white blood cells. This treatment leaves the patient temporarily without an immune system. Finally, the doctors return to the patient’s bloodstream the stem cells that had been isolated. Being in blood expedites their transformation into working immune cells.

These stem cells—sometimes called adult stem cells rather than embryonic stem cells—form a fresh army of white blood cells that’s less likely to make rogue antibodies.

“We kind of reboot the computer,” says Richard K. Burt, a physician and immunologist at Northwestern University School of Medicine in Chicago, who pioneered the therapy.

Since 1997, when Burt performed the first stem cell therapy for lupus, he and his colleagues have treated 48 people at Northwestern Memorial Hospital in Chicago. The patients all had life-threatening disease or impending organ damage and weren’t expected to improve.

No patient died from the therapy, Burt and his team report in the Feb. 1 Journal of the American Medical Association (JAMA). As of 6 months ago, after an average follow-up of 29 months, 42 of the patients were still alive. Lupus was in remission in 33 of these patients, Burt says. One patient has survived nearly 8 years.

Roughly 1.5 million people in the United States have lupus. “About 15 to 20 percent of them [become] seriously ill,” says Joan Merrill, medical director of the Lupus Foundation of America and a rheumatologist at the Oklahoma Medical Research Foundation in Oklahoma City.

Although preliminary, she says, “these new data are very exciting.”

While a stem cell transplant doesn’t necessarily represent a cure, “the therapy offered substantial benefit … to the majority of patients,” say lupus specialist Michelle Petri and hematologist Robert A. Brodsky of Johns Hopkins Medical Institutions in Baltimore in the same issue of JAMA.

Burt’s team has received Food and Drug Administration clearance to begin a large-scale trial in which randomly selected volunteers will get either the stem cell treatment or the best medication currently available.