Stem Cells Carry Hope for Lupus
Small Study Indicates Therapy Boosted Survival For the Sickest Patients
By GAUTAM NAIK
Staff Reporter of THE WALL STREET JOURNAL
February 1, 2006; Page D5
Scientists have used a form of stem-cell therapy to improve survival rates for the most severely sick lupus patients, a group that usually has no remaining treatment options.
Lupus is an autoimmune disease that attacks an individual's own organs, causing symptoms that range from swollen joints and skin rashes to severe damage to kidneys, lungs and other parts of the body.
It affects about 1.5 million people in the U.S., mainly young women. The ailment is three times more common and often more severe among African-Americans and people of Hispanic origin.
In the study of 50 people with lupus, patients received transplants of blood stem cells that originated from their own bone marrow, apparently inducing remission of the disease. Scientists found that half the patients were symptom-free and were in remission at five years, and 84% overall had survived five years. There was no untreated control group.
The findings are to be published Wednesday in the Journal of the American Medical Association.
The study was small and risky, and the results are preliminary. But its success has paved the way for a larger trial, with about 110 patients, expected to begin in a few months. The new trial would include a control group of patients.
Lupus patients are usually treated with drugs that suppress their immune systems. But for about 5% to 10% of such patients, the drugs no longer work. The latest study was launched in 1997, enrolled patients from 20 states and ran through January 2005.
The procedure was inspired by an approach sometimes tried in cancer treatment. First, bone-marrow stem cells were harvested from the patients' own blood. Then, through high doses of chemotherapy, researchers destroyed the patients' existing immune systems. Finally, they cleansed the stem cells and returned them to the bone marrow in an effort to regenerate a healthier immune system for each patient.
"It's as if your computer frizzed out and we reboot it," said Richard Burt, chief of the division of immunotherapy for autoimmune diseases at Northwestern Memorial Hospital in Chicago and lead author of the paper. He is also associate professor of medicine at Northwestern University's Feinberg School of Medicine.
A multicenter European study that also used stem-cell transplantation for lupus patients, published in 2004, reported a similar success rate. But in that trial, the methods used varied from center to center, according to Dr. Burt. And it reported a treatment-related mortality rate of 13%, compared with 2% for the U.S. study.
Nobody knows what causes lupus, but scientists believe hormones, faulty genes and environmental factors or some combination of them might trigger the malady.
Dr. Burt hopes one day to undertake a more ambitious study: transplant a sibling's stem cells into a lupus patient and see if "rebooting" the immune system with the sibling's fresh cells could actually cure the disease.
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