When most people hear the word arthritis, they think of grandma’s stiff, swollen fingers. But children get arthritis, too — a form that, like adult rheumatoid arthritis, is an autoimmune disease.

Juvenile arthritis is relatively rare, affecting just under 300,000 American children and teens. It’s so poorly understood that it’s formally known as JIA, for juvenile idiopathic arthritis — "idiopathic" loosely translates to “we don’t know why.”

But in recent years, several related classes of drugs, called biologics, have dramatically improved patient outlook.

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New Landscape, More Able-Bodied Patients

JIA causes pain and swelling in one or many joints. In some cases, the eyes and internal organs can also swell; rashes and fevers are also common. Over time, joints become deformed and degraded.

But with newer medications, these hallmarks of the disease have almost disappeared.

“When I started in this field, we’d have 30 to 40 percent [of patients] in wheelchairs,” said Dr. Patience White, a medical professor at George Washington University in Washington, D.C., and a spokesperson for the Arthritis Foundation, describing camps for children with JIA. “I don’t see wheelchairs anymore.”

“When you look at the children, the difference it makes it’s huge,” said Dr. Laura Schanberg, a pediatric rheumatologist at Duke University medical center in Durham, N.C. “If you go to the JA conference for families of children with arthritis, you can’t tell which kid in the family has the disease. In fact, the only people you can tell have had arthritis are the counselors, because they’re old enough that they had to take the older drugs.”

The new drugs are dubbed “biologics” because they’re made from active biological material, rather than chemical compounds. Many are monoclonal antibodies, or drugs that behave like human antibodies trained to attack a specific threat to the immune system. Most of the newer JIA drugs target parts of the immune system that cause inflammation.

Just last year, the FDA approved two new drugs, tocilizumab and canakinumab, to treat systemic onset JIA, one of the most difficult forms to treat. The drugs had already been in use to treat adult rheumatoid arthritis.

The biologic drugs arthritis specialists prefer have all been approved in the past six years, and already at least 1 in 3 JIA patients takes the drugs.

New Drugs Bring Unanswered Questions

But the major shift in treatment regimens has brought new challenges as well.

The new drugs are immune suppressants that carry more serious side effects than earlier JIA treatments. 

“People do die of infections when they’re on these drugs," said White. "They’re a great thing but they do dampen your immune response.” 

Short-term side effects, though usually considered a worthwhile tradeoff, raise questions about longer-term side effects. Because the biologic drugs are new, no one knows what consequences they may have for young patients after 10 or 20 years. The immune system plays a role keeping cancer at bay, for instance, and adults with rheumatoid arthritis face elevated risks of some types cancer. 

The Childhood Arthritis and Rheumatology Research Alliance, which Schanberg now leads, is building a registry of patients taking the new drugs to provide a robust data set to track any potential long-term risks.

Even with unanswered questions, Schanberg is adamant about the drugs’ benefits.

“I have concerns, but I know what happens if they don’t use the drugs. You have to keep things in perspective,” she said.

Perhaps the greatest problem is that for some patients who are good candidates for these medications, the drugs remain out of reach. Most family doctors don’t know how to use biologics, according to White, because JIA is rare and the drugs are new.

Early intervention is key to protect joints from permanent damage, so families need to take affected children to a specialist right away. Yet, in at least 11 states there isn’t a single pediatric rheumatologist, according to Arthritis Foundation data.

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Then there’s the issue of insurance coverage. Many insurers categorize biologics as specialty drugs, leaving families with high out-of-pocket costs.

“It’s a significant issue for families. To try to get these drugs for their kids, some have tried to sell their homes,” White said. 

The Arthritis Foundation is lobbying for expanded access to the new drugs.