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Juvenile arthritis prompts study
by Holly Auer
Of the Post and Courier Staff
May 9, 2005
'Target inhibitors' for immune system show promise
Eight-year-old Sarah Windham has hardly known life without pain. Doctors diagnosed her arthritis when she was just 15 months old. The years since have meant a cocktail of medications to keep her right knee from flaring up, and missing weeks of school for doctors appointments and tests.
When it comes, the pain is so intense Sarah doesn't yet know the words to truly describe it. Once, it lasted a whole year.
When Vioxx was pulled from the market last fall, a seemingly unlikely group of arthritis patients — children like Sarah — felt its disappearance keenly.
Although most people develop the disease when they're older, 285,000 American children with it face a lifetime of sometimes crippling joint pain. The anti-inflammatory drug Vioxx helped many of them. Now, scientists' quest to find better arthritis treatments is especially urgent since medications to control arthritis and other autoimmune diseases have a host of worrisome side effects.
Poorly managed arthritis tends to consign children to a life of missing school, sleep, sports and other activities.
"In the past, children with arthritis used to sit on the sidelines as if life was passing them by," said Richard Silver, a rheumatologist and professor of medicine and pediatrics at the Medical University of South Carolina. "Fortunately, the outlook for them is pretty good now."
Studies under way at MUSC are honing the science of "target inhibitors," which act on the parts of the immune system that misfire and spur diseases such as arthritis, lupus and multiple sclerosis.
In a sense, researchers are working on "smart" drugs. The drugs know just where in the body to go, minimizing damage to other parts of the body, said Dr. Stephen Tomlinson, a professor of microbiology and immunology who runs studies on target inhibitors in mice with arthritis.
The next step will be to conduct research that brings his work in the lab to patients, likely through long-lasting intravenous medications.
Medicines used to treat autoimmune diseases today work, but they tend to damage the immune system, leaving kids open to more infections and even cancer. Long-term steroid use can affect a child's growth, and it has been linked to bone problems and cataracts.
"Instead of a hydrogen bomb, we want to be using a cruise missile to treat," Silver said. "When you knock the inflammation out globally, you're going to get other problems."
In a perplexing twist, the side effects of current medicines sometimes mimic symptoms of the disease they're being used to treat. When 12-year-old Jordan Price runs a fever, for instance, his mother can't tell whether that's his lupus talking or an infection he caught at school because the medicines weakened his immunity.
"I never really know whether to call the pediatrician or the rheumatologist," said LaVerne Price, who also suffers from lupus. "It's a trying thing for him, to never know what's going on. But he's a fighter."
Price knows the costs of taking the drugs, too. Over time, her lupus medicines caused her bones to deteriorate so badly she had to have a hip replacement and had to quit her teaching job. When her son, a sixth-grader at West Ashley Intermediate School, looks to his future, she wants him to see a life that isn't ruled by illness.
"Childhood is kind of a setup for how bad these diseases will be later on," said Dr. Bernard Maria, executive director of MUSC's Children's Research Institute, which houses the research programs for rheumatology and autoimmune disease. "If we can understand its origins in childhood, we're very likely to contribute to new treatments that are much more honed to the biological process."
The hope is that these types of autoimmune diseases could be identified from their earliest moments and be thwarted with targeted treatment before they truly take hold in the body.
Sarah's parents try to salve the ache with fun times, like drawing early-morning bubble baths to ease her pain with heat before school. Despite it all, the Summerville second-grader is a stand-out athlete, excelling at fast-pitch softball and racking up awards in ice skating.
She's lucky, all things considered. Her parents saw other children curled into wheelchairs at a juvenile arthritis conference they attended a few years ago, and it made them weep.
"It's terrible, what happens to these kids," said her mother, Kathy Windham. "We need to be doing so much more research on kids, because this isn't just about making drugs for the elderly."
Tomlinson's work likely will bring treatments to market to help children like Sarah, but that won't help the shortage of doctors to plan their care. Nationwide, only about 15 people finish their training in pediatric rheumatology each year, and at least five of those are foreign doctors who head back to their native countries to practice, Silver said. He figures that to properly treat every child with rheumatic diseases in the United States, the country needs another 150 to 200 specialists.
This winter, after she'd gone more than a year without a flare-up and had a chunk of time off her medicines, Sarah's family had started believing she'd kicked the disease for good. Then in January, it came back. Sarah wound up in the hospital getting a steroid injection and having fluid drained from her knee, which had swollen so it looked like she had a baseball plopped on top of it.
Kathy Windham worries about the steroids' long-range effect on Sarah's body. Another medication she took caused trouble with her liver. Windham is certain the drugs have stunted her daughter's size; until last year, Sarah was always the tiniest one in her class, still wearing toddler-sized shorts in first grade.
During the time she was off the medicine, though, Sarah's appetite bounced back, and she put on 13 pounds in 10 months. When she ice skates and lands her jumps on her bad leg, her mother can hardly believe everything she has overcome.
"She's incredible," Windham said.
"Watching her just makes you appreciate life so much more."
Holly Auer covers health and medicine. She can be reached at (843) 937-5560 or hauer@postandcourier.com.
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