New Drug Therapy for Juvenile Rheumatoid Arthritis

It's hard to believe that not long ago, a seven-year-old Wisconsin girl was in such constant pain that she used a tricycle to move between classrooms at school, she could not walk across the street or even use coloring books.

Once ravaged by juvenile rheumatoid arthritis (JRA), the girl now rides a bicycle, walks to the park and back on her own, rough-houses with her older brother, and even runs and skips like a little dervish.

It's all thanks to a new drug therapy she received under the care of James Nocton, MD, assistant professor of pediatrics at the Medical College of Wisconsin. Dr. Nocton is based at Children's Hospital of Wisconsin, an affiliate of MCW.

The little girl was one of the first 50 juvenile rheumatoid arthritis patients in the country to try Enbrel, a genetically engineered human protein which seems to halt progress of the disease in some patients. She began taking Enbrel as part of a national clinical trial by the U.S. Food and Drug Administration and the drug's manufacturer, Immunex Corp. Children's Hospital is one of nine participating test sites.

A rheumatologist at Children's Hospital, Dr. Nocton helped run the Enbrel study in which the girl participated.

At Children's Hospital, eight patients began receiving the drug in the fall of 1997, with six showing dramatic improvement, and the seven-year-old girl doing the best of all. Because Enbrel is so new, the long-term effects are unknown and remain somewhat of a concern for her family. But the results have been stunning so far.

The girl takes Enbrel shots twice a week. Enbrel works by neutralizing a protein called tumor necrosis factor (TNF). An essential component of the immune system, TNF seeks out and destroys diseased cells. But when the body makes too much TNF, it is very destructive and is a suspected agent in many diseases from cancer to arthritis.

At just 15 months of age, the girl developed systemic-onset JRA, which means the disease causes illness, with fevers, fatigue and poor appetite, and may affect any joint in the body.

"It is the most painful form of JRA," said Dr. Nocton. "Most of the time it eventually goes away after a few months or several years, but in about a quarter of these kids their arthritis persists and continues to get worse. [The seven-year-old girl] falls into that category."

Treatment of JRA at Children's Hospital is a team approach that includes specialists from occupational and physical therapists to nutritionists, gastroenterologists, nurses and social workers.

Enbrel does not cure arthritis, but can halt its progress and prevent joints from deteriorating. Many children will in essence outgrow their arthritis as it goes into remission, usually in early adulthood. While this seven-year-old may always have some joint stiffness, she is regaining the strength and coordination she lost from years of living in pain. She is currently looking forward to being a nurse someday like her mother.

For more information or to set up an appointment, please call 414-266-6700.

MCW Health News presents up-to-date information on patient care and medical research by the physicians of the Medical College of Wisconsin.