New drug curbs rheumatoid arthritis in adults, children

A novel drug that takes a narrow approach to quelling inflammation might be just the ticket for people with rheumatoid arthritis who react poorly to broad-spectrum medications, a new trial from Austria indicates.

The drug, called tocilizumab, might also offer a reprieve for children with hard-to-treat juvenile idiopathic arthritis (JIA), formerly known as juvenile rheumatoid arthritis, a Japanese study shows.

Tocilizumab specifically blocks the action of the inflammatory protein interleukin-6, which shows up in excessive amounts in joints of rheumatoid arthritis patients.

Josef Smolen of the Medical University of Vienna and his colleagues enlisted 622 people with moderate to severe rheumatoid arthritis. All had failed to improve on methotrexate, a frontline drug. Two-thirds of the patients received intravenous infusions of tocilizumab every 4 weeks for 24 weeks, while the others received placebo infusions.

Those getting the drug reported significantly less joint pain and stiffness as well as a better quality of life, the researchers report in the March 22 Lancet.

Meanwhile, Shumpei Yokota of Yokohama City University in Japan and his team enlisted 56 children with JIA, which is marked by chronic arthritis, fever, rash, and other symptoms. All the patients received tocilizumab for 6 weeks. At that point, the researchers randomly assigned 43 who were faring the best to get the drug or placebo for 12 more weeks. Those getting tocilizumab were five times as likely as those on placebo to maintain improvements in symptoms, Yokota and his colleagues report in the same Lancet issue.

Tim Bongartz, a rheumatologist at the Mayo Clinic in Rochester, Minn., says the findings might clear the way for regulatory approval—although he would prefer to see more studies to reveal any long-term side effects tocilizumab might cause.

In any case, he says, “it’s a huge step forward,” particularly for children with JIA, who have access to few approved medications.