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BrainChild-04 Study Is Enrolling Patients With Brain and Spinal Cord Tumors

June 7, 2023

About BrainChild-04

In May 2023 Seattle Children’s began enrolling patients in a new study of a novel treatment for certain types of brain and spinal cord tumors in children and young adults. These include diffuse intrinsic pontine glioma (DIPG),  diffuse midline glioma (DMG) and other recurrent or refractory central nervous system tumors that carry a poor prognosis with limited survival.

The BrainChild-04 study is investigating Seattle Children’s Therapeutics’ first CAR T-cell product that targets four antigens simultaneously: B7-H3, EGFR806, HER2 and IL13-zetakine.  This is the first known CAR T-cell product in the world to target four antigens at the same time by delivering CAR T cells directly to the brain.

A long-standing obstacle to successfully treating brain tumors has been the blood-brain barrier, which stops therapeutic medications from reaching their intended targets.

Up to 72 participants between the ages of 1 and 26 are expected to enroll in this phase 1 clinical trial.

“We are working hard to provide new therapies and learn as much as possible to bring hope to patients today and in the future,” says study chair Dr. Nicholas Vitanza.

Tackling the toughest tumors

DIPG and DMG are childhood tumors for which there is no known cure. Most children are diagnosed around age 6 or 7, and surgery is not an option for these tumors due to their location in the brain. The average survival is only 11 months. Other central nervous system tumors included in this study are pineoblastoma, ATRT, ETMR medulloblastoma and ependymoma.

About 5,000 kids in the United States are diagnosed with brain and spinal cord tumors each year.

Seattle Children’s is at the forefront of advancing understanding of brain tumor treatments

Five years ago, researchers had never delivered CAR T cells directly to a child’s brain. Today research at Seattle Children’s is showing that it’s both possible and tolerable. More than 400 intracranial doses have been delivered to children, teens and young adults enrolled in the BrainChild clinical trials to date.

Seattle Children’s has one of the largest pediatric cellular immunotherapy programs in the world and has enrolled more than 500 patients.

Seattle Children’s Therapeutics builds upon what is learned in each trial to create next-generation therapies. Since 2012, the team has designed, manufactured and launched a robust portfolio of cellular immunotherapy clinical trials for childhood cancer in the areas of leukemia, lymphoma, brain and spinal cord tumors and solid tumors.