Children’s Wisconsin clinical research teams recently launched a phase 3 trial, led locally by Angela Steineck, MD, Assistant Professor of Hematology and Oncology-Pediatrics, to test the effects of adding an immunotherapy, called dinutuximab, to initial chemotherapy in children with newly diagnosed high-risk neuroblastoma. Dr. Steineck explained this novel approach may target cancer cells more effectively, potentially reducing relapse and improving survival for patients experiencing one of the most aggressive pediatric cancers.
“Dinutuximab works by binding to GD2, a molecule on the surface of neuroblastoma cells,” said Dr. Steineck. “This flags the cancer cells for destruction by the immune system, activating natural killer cells and macrophages to attack the tumor. By introducing dinutuximab during induction therapy, we combine its immune-boosting effects with chemotherapy’s ability to directly damage the tumor.”
Neuroblastoma is the most common extracranial solid tumor in children, with nearly 800 new cases diagnosed annually in the United States. High-risk neuroblastoma is especially aggressive, with a five-year survival rate of approximately 50%. Standard chemotherapy can shrink tumors but often leaves residual disease that can lead to relapse. Adding dinutuximab to initial therapy may overcome this challenge by activating the immune system to eliminate microscopic cancer cells, potentially making treatment more effective.
Children’s Wisconsin is a leader in pediatric clinical research that offers specialized expertise and advanced therapies for children facing aggressive diseases—it was recently named among the nation’s best in U.S. News & World Report’s 2024-25 Best Children’s Hospitals report. Its MACC Fund Center for Cancer and Blood Disorders is at the forefront of innovation, providing access to groundbreaking clinical trials that explore the latest treatments. Through its involvement in early-phase trials, Children’s Wisconsin has pioneered personalized approaches to care, such as novel immunotherapies and precision medicine strategies.
“These efforts ensure that families navigating complex and high-risk diagnoses receive tailored, state-of-the-art treatments,” added Dr. Steineck. “We’re hopeful this trial will transform outcomes, giving children with high-risk neuroblastoma a better chance at long-term health and recovery.”
The National Cancer Institute-sponsored study aims to enroll 478 participants across all sites, including 10 at the Children’s Wisconsin site.