Could cancer-fighting therapy be used to treat young lupus patients? Seattle Children's study seeks answers

The U.S. Food and Drug Administration has given Seattle Children’s the green light to conduct a clinical trial for a drug that researchers hope can help fight pediatric lupus.

The drug known as chimeric antigen receptor-T cell therapy, which can help destroy disease-causing cells in cancer patients, will be put to the test for lupus patients for the first time, a Seattle Children's spokesperson said in a statement.

Pediatric lupus is a rare autoimmune disease affecting multiple organs like the brain, kidneys, and lungs. Physicians don't know exactly what causes the disease, but some experts have hypothesized that the onset is related to genetic or environmental influences, or even differences in sex.

Women and girls between the ages of 15 and 44 make up 90% of the roughly 1.5 million cases of lupus in the U.S. The Seattle Children’s clinical trial will, in part, be focused on understanding that disproportionality.

“This is an important clinical trial because we don't know yet exactly [why] particularly girls can be affected by lupus,” said Dr. Vittorio Gallo, chief scientific officer at the Seattle Children’s Research Institute. He added that 20% of all American lupus patients are children.

Seattle Children’s, which sees about 20 pediatric lupus patients each year, is the primary pediatric referral center for children throughout Washington, Wyoming, Alaska, Montana, and Idaho with the disease.

“Children who are younger than 5, which is pretty rare, [are] typically among the sickest among the lupus patients,” Gallo said.

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Research shows this may be due largely to young children not having completely developed immune systems. How injurious the effects of the disease are on children, “depends on the onset of the disease,” Gallo explained.

“If vital organs are affected, then these children have to live for the rest of their lives with the impact of the disease on these organs,” he added.

Gallo said symptoms of lupus, which include “fatigue, loss of appetite, weight loss, muscle aches, swollen joints, and fever,” can be misleading, as they are associated with many other medical conditions.

“So initially, it can be difficult to diagnose, which means that it delays pharmacological treatment, and therefore — particularly in populations that have reduced access to health care — the delayed diagnosis can cause significant disparities in the treatment of the disease,” Gallo added.

He said the trial will also explore why there is a higher incidence of pediatric lupus in African American, Native American, and Latino populations. Research shows African American and Native American patients are three times more likely to have lupus compared to white patients, while Latino patients are twice as likely. But not only are the rates of the disease higher among these populations — the symptoms and prognoses are more severe.

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“[Effects of the disease are] nine times worse in African American patients than in white patients,” Gallo said. “And the progression to kidney failure, which is one outcome in children, is nine times higher in African American patients compared to whites.”

Gallo said researchers are trying to tease out how socioeconomic status, poverty, access to health care, along with the timing of diagnosis impact patients’ longevity.

“The trial will start later this year, and will be longitudinal, meaning physicians will have to follow up with patients for a few years to look at improvement of their condition,” Gallo said.

Update notice, Friday March 15, 2024 at 3:10 p.m.: This story has been updated to clarify that the clinical trial will test chimeric antigen receptor-T cell therapy for pediatric lupus patients.