In 2010, Dr. Yu led a randomized phase III trial that demonstrated that immunotherapy with dinutuximab, a chimeric anti- GD2 mAb, GM-CSF, and IL2 improved survival for children with high-risk neuroblastoma that had responded to induction and consolidation therapy. These results served as the basis for FDA approval of dinutuximab. In a long-term follow-up study, published in 2021, Dr. Yu led the clinical research that demonstrates that after 5 years, immunotherapy with dinutuximab in combination with cytokines continues to be associated with an improvement in survival in children with high-risk neuroblastoma. However, the magnitude of the survival benefit for patients randomized to immunotherapy has decreased over time due to late relapses, demonstrating the necessity to associated biomarkers that can help identify patients most likely to benefit from immunotherapy.
Kaplan–Meier survival curves of EFS (event free survival) for patients who are ≥18 months old at diagnosis with International Neuroblastoma Staging System (INSS) stage 4 disease (n=158), by randomized treatment arm on COG study ANBL0032. EFS was statistically significantly higher in patients assigned to immunotherapy compared with those assigned to isotretinoin. Clin Cancer Res. 2021 Apr 15;27(8):2179-2189