10/27/08 Researchers have discovered a new biologic drug that may help the most stubborn and debilitating cases of gout.
The news was released at the 2008 American College of Rheumatology Scientific Meeting in San Francisco.
Scientists at Duke University in Durham, N.C., have recently completed stage III clinical trials of a gout medication called pegloticase, brand name Puricase (ed. note: brand name later changed to Krystexxa) – a time-released protein given by intravenous infusion that helps to break down excess levels of uric acid. Uric acid is a metabolic byproduct that, at high levels, forms crystals that deposit in the joints, causing pain, swelling, and deformity.
If federal regulators approve the drug in the coming months, it may help a small but significant group of people who have not yet had a way to control their gout.
“This patient group [represents] one of the challenges that rheumatologists have faced for a long time,” said Dr. John Sundy, associate professor of medicine at Duke. “They are very severely affected.”
Dr. Sundy and his team randomly assigned 212 participants with “treatment-failure” gout to one of three groups: one that took a lower dose of the drug, one a higher dose, and one a placebo. Neither the study volunteers nor their doctors knew who was getting the new gout drug or the placebo.
People qualified for the study if they had a history of more than three gout flares in 18 months or if they had at least one joint deformed by the disease; if they had high levels of uric acid in their blood; and if standard drug treatment with the current “gold standard” gout medication, allopurinol, had not worked.
About 40 percent of participants treated with pegloticase saw their blood levels of uric acid drop “within hours” of starting the drug, Dr. Sundy said. The reduction in uric acid translated into significant improvements in physical functioning and a drop in the number of tender and deformed joints in the pegloticase groups as compared to the placebo group.
There was no significant difference in the number of gout flares, however, between the pegloticase groups and the placebo group.
The new gout drug also had significant drawbacks, Dr. Sundy said.
Infusion reactions occurred in 26 percent of patients treated with the lower dose and in 40 percent of patients treated with the higher dose of the drug as compared to just 5 percent of participants who got the placebo. Slightly more than one in four study participants on pegloticase suffered from other serious adverse events compared to just one in 10 of those who took the placebo.
Because of the significant side effects, Dr. Sundy stressed that if the new gout drug is approved in the coming months, pegloticase would likely be an option for only the worst gout cases.
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