Stem-Cell Transplants: A Cure for Arthritis?

Recently, Good Morning America featured a young girl with juvenile rheumatoid arthritis who had been treated with a stem-cell transplant, and the same procedure was subject of a study published in the medical journal The Lancet reporting successful treatment of people with lupus. Stem-cell transplants have been studied in Europe, Austrailia and the United States as a potential treatment for a several forms of arthritis, including juvenile rheumatoid arthritis, lupus, rheumatoid arthritis and scleroderma. However, while this therapy appears to be promising, it isn't for everyone. The Arthritis Foundation wanted to give you an update on this exciting treatment.

What is a stem-cell transplant?

A stem-cell transplant is a procedure where bone marrow is extracted from either the person with the disease, or a healthy donor. The transplants done to date in people with arthritis have only involved bone marrow cells taken from the person with arthritis – so-called “autologous” stem-cell transplantation. Most of the bone marrow cells are removed leaving predominately “stem cells,” or immature bone marrow cells that have the potential to grow, divide and develop.

Meanwhile, the person undergoing the transplant is put through a “cleansing system” to eliminate cells in the bone marrow in the body. This cleansing usually is achieved through high-dose chemotherapy or radiation treatment that dramatically suppresses the immune system. When this is done, the purified stem cells from the extraction are injected back into the body where they, hopefully, will repopulate the marrow with healthy cells, causing a complete remission.

Can I have this procedure done?

This is a risky procedure and is only performed on people with severe, life-threatening arthritis who have failed with all other standard therapy. In several meetings dedicated to stem-cell transplantation in children with rheumatic diseases, pediatric rheumatologists and bone marrow specialists from North America and Europe worked to develop guidelines for deciding who should be considered a candidate for this treatment, details about the preparation of both the stem cells and the patients, and information to be gathered before and after the transplant so as to better understand the impact of the procedure on the person. Currently stem-cell transplantation is considered an investigational procedure, and is only preformed in specialized centers participating in research studies of the procedure.

What are the risks?

A major consequence of stem-cell transplants is suppression of the immune system, which means the body's defense mechanisms are completely wiped out. Therefore, the common cold or a normally harmless virus could lead to a serious, and even life-threatening, infection. To ward against this, hospitalization is required until all treatment is completed, but this doesn't insure protection against infection or other complications.

In addition, there is a risk that the transplanted cells will not repopulate successfully. This could mean that you can be left with no or a partially effective immune system.

Because of these risks, death occurs in five to 15 percent of all stem-cell transplants.

How long does it take?

You can expect to spend between several weeks to several months in the hospital, depending on how your body reacts, complications that may arise and, most importantly, how quickly the stem cells develop into an adequate immune system.

Will this cure me?

Calling a successful stem-cell transplant a cure is still under debate. “I would say that with the information we have at this time, stem-cell transplantation may have been a cure for some of the patients,” says Daniel Lovell, MD, a pediatric rheumatologist at Children's Hospital Medical Center in Cincinnati, Ohio. While there is often no evidence of the primary disease after a transplant, Dr. Lovell feels that the procedure needs more long-term follow-up before saying that it is definitely a cure. He points out that some of the successful transplant recipients have experienced a recurrence of disease, although the activity was not as severe as it was before.

“This is very high-tech, high-risk medicine,” Dr. Lovell emphasizes. “It is for people who have no other options.”