1st CAR T-cell trial in children with lupus in US gets FDA go-ahead

Seattle Children's plans to start study in children with SLE this summer

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by Margarida Maia |

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Seattle Children’s has been cleared by the U.S. Food and Drug Administration (FDA) to start the nation’s first clinical trial testing CAR T-cell therapy in children with systemic lupus erythematosus, the most common form of lupus.

The trial, Reversing Autoimmunity through Cell Therapy (REACT-01), is targeted for a summer launch. Seattle Children’s Therapeutics, a nonprofit that’s been running immunotherapy trials for children, will lead and administer the study.

“Seattle Children’s is excited to launch this first-in-the-country trial that has the potential to offer life-changing relief for patients,” Vittorio Gallo, PhD, senior vice president and chief scientific officer at Seattle Children’s, said in a hospital press release.

“We are committed to utilizing research to pioneer breakthrough therapies and provide access to cutting-edge clinical trials in pediatric healthcare,” Gallo added.

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Autoimmune attacks in lupus are driven by self-reactive antibodies that B-cells, a type of immune cell, mistakenly produce against the body’s own tissues. In about 20% of patients, lupus symptoms begin in childhood.

Lupus symptoms tend to be worse in children, for reasons that are not fully understood, according to Seattle Children’s. Further, the autoimmune condition also tends to be more severe for historically disadvantaged patients, in many cases Black or Latino community members.

Treatment for lupus often includes the use of immunosuppressants to keep the immune system in check. However, many patients don’t respond to such treatments or may experience side effects.

Most of those who do respond to medication have to stay on it for the rest of their lives to stay in remission.

“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 Shaun Jackson, MD, PhD, a pediatrician at Seattle Children’s Hospital and the trial’s principal investigator.

The trial is expected to enroll children of all ages.

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.

CAR T-cell therapy involves collecting a patient’s own T-cells. It’s typically done by passing the patient’s blood through a machine that can separate T-cells, a type of immune cell, from the remaining components of blood. The process can take a few hours.

The isolated T-cells are sent to a lab, where they are genetically engineered to be equipped with a chimeric antigen receptor (CAR) that recognizes a specific target of interest. In the case of CAR T-cell therapies intended to treat autoimmune diseases such as lupus, that target is usually a protein found on the surface of B-cells.

The modified T-cells are then infused back to the patient. Once in the bloodstream, they are expected to home in on the disease-causing B-cells and destroy them, easing lupus symptoms.