Seattle Children’s has received FDA authorization to launch the first chimeric antigen receptor (CAR) T-cell clinical trial in the U.S. for children under 18 with the most common form of lupus.
Systemic lupus erythematosus is a chronic autoimmune disorder that affects multiple organ systems, resulting in significant morbidity and mortality. Approximately 20% of lupus patients have disease onset during childhood, and children with lupus are at particular risk for severe disease and worse long-term outcomes.
For reasons that are not well understood, the incidence and severity of lupus is also increased in historically disadvantaged patient populations in the U.S., including Black and Latino communities.
Despite recent advances in lupus management, many patients have lupus that is resistant to current treatment regimens. Even among treatment-responsive subjects, available medications need to be taken chronically with little expectation of durable remission off therapy.
“Seattle Children’s is excited to launch this first-in-the-country trial that has the potential to offer life-changing relief for patients,” said Dr. Vittorio Gallo, senior vice president and chief scientific officer at Seattle Children’s. “We are committed to utilizing research to pioneer breakthrough therapies and provide access to cutting-edge clinical trials in pediatric healthcare.”
The trial, which will be known as the Reversing Autoimmunity through Cell Therapy, or REACT-01 study, will be led and administered by Seattle Children’s Therapeutics, a novel non-profit therapeutics development enterprise, devoted to envisioning and testing next-generation cell and gene therapies for pediatric diseases so children have the medicines they deserve.
“Our hope is that this therapy holds the potential to put patients with systemic lupus erythematosus into true remission without the need for ongoing medications,” said Dr. Shaun Jackson, principal investigator for the study.
The target opening date for the trial is this upcoming summer.
