Unituxin (dinutuximab)

Recent advances in immunotherapy have reshaped the treatment landscape, with anti-GD2 monoclonal antibodies such as dinutuximab and naxitamab demonstrating significant clinical benefit, especially when combined with granulocyte-macrophage colony-stimulating factor (GM-CSF)...This review synthesizes current evidence on immunotherapeutic strategies in neuroblastoma, highlighting resistance pathways, biomarker-driven approaches, and the evolving clinical trial landscape. Future directions emphasize personalized, biomarker-guided immunotherapy to improve efficacy, reduce toxicity, and establish durable, curative outcomes for children with neuroblastoma.

P1, N=11, Active, not recruiting, Children's Hospital Los Angeles | Recruiting --> Active, not recruiting

For patients ≥547 days old, any age cut-off ≤40 months discriminated younger (superior EFS/OS) versus older patients; no cut-off was optimal. OCAYA diagnosed 2010-2022 (post-immunotherapy era) had superior outcomes versus 2003-2009. Stratification by comprehensive molecular biomarkers will likely best inform novel therapeutic strategies for OCAYA.

P1, N=24, Recruiting, Emory University | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Dec 2025 --> Dec 2026

P2, N=118, Not yet recruiting, New Approaches to Neuroblastoma Therapy Consortium

Dinutuximab, approved for high-risk neuroblastoma, improved event-free survival when combined with cytokines with standard therapy alone (NCT00026312). Similarly, Naxitamab, in combination with GM-CSF, achieved an overall response rate in relapsed/refractory neuroblastoma (NCT03363373)...Additionally, Racotumomab (anti-NeuGcGM3) demonstrated a survival benefit in advanced non-small cell lung cancer, extending median overall survival compared to placebo (NCT01240447). Despite these advances, challenges remain in improving patient selection, reducing off-target toxicities such as neuropathic pain, and addressing resistance mechanisms. This review examines the latest developments in ganglioside-mediated cancer therapy, emphasizing current clinical outcomes, highlighting the need for more precise targeting approaches, and exploring rational combination strategies to enhance therapeutic efficacy.

P2, N=62, Recruiting, New Approaches to Neuroblastoma Therapy Consortium | Active, not recruiting --> Recruiting

P2, N=41, Active, not recruiting, Children's Oncology Group | Trial completion date: Sep 2025 --> Sep 2027

P2, N=42, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Sep 2025 --> Sep 2026

Immunotherapy further improved survival rates that included monoclonal antibody (Anti-GD2 MoAb/Dinutuximab) and cytokines (ie, IL-2 and GM-CSF, [33-37])...This allowed them to produce a CAR that targets a tumor-specific antigen. Following preparatory chemotherapy, the CAR T cell product is administered into the patient.

P1/2, N=47, Completed, Eli Lilly and Company | Active, not recruiting --> Completed | Trial completion date: Dec 2025 --> Aug 2025

Profile C was found only in cell lines of the mesenchymal subtype. These findings support further investigation of GG composition and associated enzyme expression as potential biomarkers for risk stratification and treatment response in NBL.