New studies testing the effectiveness of two biologics in children with systemic juvenile idiopathic arthritis (sJIA) have found that both drugs – tocilizumab (Actemra) and canakinumab (Ilaris) – result in major improvements. In some cases, kids felt better within a day.

“The results are thrilling. They are dramatic. They will be game changers for kids with sJIA,” says Daniel J. Lovell, MD, co-author of the studies and associate director of the division of rheumatology at Cincinnati Children's Hospital Medical Center.

Actemra was approved by the U.S. Food and Drug Administration (FDA) in 2011 for sJIA, a potentially life-threatening form of juvenile arthritis that causes inflammation in joints and organs, as well as fevers, rash and fatigue. But Dr. Lovell says that some pediatric rheumatologists have been waiting to see the results of larger clinical trials before prescribing Actemra in lieu of older drugs.

Ilaris is not FDA-approved for sJIA, but it is prescribed for the condition “off-label.” Novartis, the drug's manufacturer, says data from the trials form "the basis for worldwide regulatory submissions," which is "on track" in the U.S.

The new research – carried out by two international research networks, the Pediatric Rheumatology Collaborative Study Group and the Paediatric Rheumatology International Trials Organisation – was published recently in two articles in The New England Journal of Medicine. The first article detailed a randomized trial of tocilizumab, a drug that inhibits interleukin-6 (IL-6), a cytokine – or molecule – involved in immune and inflammatory processes. The study found that two-thirds of patients taking tocilizumab had significant improvement after just one dose. After a year on the drug, 80 percent of patients had no fever and an improvement in symptoms of at least 70 percent.

The second article detailed two randomized trials of canakinumab, which targets a different cytokine, interleukin-1 (IL-1), also involved in immune and inflammatory processes. The first trial, which lasted nearly a month, showed 84 percent of participants had marked improvement in symptoms after two weeks on the medication. The second (longer) trial showed 74 percent had no flares compared to those receiving placebo.

“With either treatment the systemic features resolved very quickly – from a few hours to a few days. In many cases you start the medicine in the morning and come back in the afternoon and the kids are feeling better. It’s extraordinary,” Dr. Lovell says. “It’s like these two agents are designer drugs for sJIA because the disease is driven by either interleukin-1 or interleukin-6, or a combination of the two. You can block either one and for 80 percent of these kids, it’s truly dramatically different. Their lives are profoundly different.”