An exciting new treatment makes it’s way to young patients—and it’s all thanks to Baylor research.
For parents of children with systemic-onset juvenile idiopathic arthritis (sJIA), the normalcy of a worry-free childhood is often traded for years of extensive testing, difficult treatments and long hospitalizations.
But a new treatment discovered by Baylor researchers is changing all that.
A Devastating Disease
SJAI affects approximately 30,000 children in the United States alone, and accounts for about 10 percent of all childhood arthritis cases.
“Children with sJIA suffer from pain, limited mobility and potential joint damage that may result in long-term disability,” explains Virginia Pascual, M.D., director for the Centers of Inflammation and Genomics at the Baylor Institute for Immunology Research (BIIR), a part of the Baylor Research Institute (BRI).
As a pediatric rheumatologist, Dr. Pascual had seen the results of traditional sJIA treatments like steroids, which caused “significant morbidity, including vertebral compressions fractures, cataracts and severe growth retardation.” So in 2005, she piloted a clinical trial at BIIR using an exciting new treatment approach.
“When we combined healthy white blood cells with a patient’s own serum (plasma with clotting elements removed), we observed the activation of inflammatory molecules that could explain the development of arthritis,” Dr. Pascual explains. “So we attempted to block these molecules with drugs that were already available.”
It worked. “Seven of the nine children we treated went into full remission,” she says. The results of the study, which utilized a drug called anakinra to achieve its results, were published in The Journal of Experimental Medicine—but more extensive trials would be needed to prove the treatment’s effectiveness.
The Real McCoy
In 2011, BIIR conducted the first randomized clinical trial using anakinra in SJIA patients in collaboration with the Necker Hospital in Paris. And in December 2012, the results of two large trials with hundreds of participants from multiple centers across the world were published in The New England Journal of Medicine.
“In every case, up to 70 percent of patients went into remission,” Dr. Pascual says.
The results confirmed Baylor researchers’ initial observations—they had discovered a new treatment. And today, it is being used worldwide.
“Because of our discovery, thousands of children with sJIA will go into full remission,” she says. “We’re incredibly proud of that.”
And the pioneering work doesn’t end there. Today, BIIR researchers are using a similar approach to find new treatments for lupus.
“We are excited about ongoing clinical trials, also based on our findings, that may bring about similar success for patients with lupus in the future,” Dr. Pascual says
Want to get involved? Visit BaylorHealth.com/AdvancingMedicine today to learn about research trials at Baylor seeking participants.