Seattle Children's has commenced enrollment for the BrainChild-04 study, a Phase 1 clinical trial investigating a novel CAR T-cell therapy for children and young adults with certain brain and spinal cord tumors. This innovative treatment targets four antigens—B7-H3, EGFR806, HER2, and IL13-zetakine—simultaneously, marking the first known CAR T-cell product worldwide to employ such a multi-targeted approach. The study focuses on tumors with poor prognoses, including diffuse intrinsic pontine glioma (DIPG), diffuse midline glioma (DMG), and other recurrent or refractory central nervous system (CNS) tumors.
Addressing Unmet Needs in Pediatric Brain Tumors
DIPG and DMG are particularly devastating childhood tumors for which there is currently no cure. Typically diagnosed around the age of 6 or 7, these tumors are inoperable due to their location in the brainstem, leading to an average survival of only 11 months. The BrainChild-04 study also includes other CNS tumors such as pineoblastoma, ATRT, ETMR medulloblastoma, and ependymoma, representing a significant unmet medical need for these patients. Approximately 5,000 children in the United States are diagnosed with brain and spinal cord tumors each year, underscoring the urgency for new therapeutic options.
Overcoming the Blood-Brain Barrier
A major challenge in treating brain tumors has been the blood-brain barrier, which restricts the passage of therapeutic medications to the tumor site. The BrainChild-04 study addresses this obstacle by delivering CAR T-cells directly to the brain, ensuring that the therapy reaches its intended targets. This direct delivery method builds upon Seattle Children's extensive experience in intracranial CAR T-cell administration, with over 400 doses delivered to children, teens, and young adults enrolled in the BrainChild clinical trials to date.
Study Design and Patient Population
The BrainChild-04 trial aims to enroll up to 72 participants between the ages of 1 and 26. According to study chair Dr. Nicholas Vitanza, the team is dedicated to providing new therapies and maximizing learning to bring hope to patients both now and in the future. Seattle Children's Therapeutics is leveraging the knowledge gained from previous trials to develop next-generation therapies. Since 2012, the team has designed, manufactured, and launched a robust portfolio of cellular immunotherapy clinical trials for childhood cancer, spanning leukemia, lymphoma, brain and spinal cord tumors, and solid tumors.
Seattle Children's Leadership in Cellular Immunotherapy
Seattle Children's boasts one of the largest pediatric cellular immunotherapy programs globally, having enrolled more than 500 patients. This trial represents a significant step forward in the treatment of pediatric brain tumors, offering a novel approach to overcome the challenges of the blood-brain barrier and target multiple antigens simultaneously.
