Danyelza (naxitamab-gqgk)

Naxitamb + GM-CSF is an attractive option for primary refractory osteomedullary disease, including in patients with a high disease burden.

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.

P1/2, N=31, Recruiting, M.D. Anderson Cancer Center | Trial completion date: Nov 2030 --> Jul 2030

P1/2, N=31, Recruiting, M.D. Anderson Cancer Center | Not yet recruiting --> Recruiting

Ongoing clinical studies and expanded global use continue to evaluate its safety and efficacy, to optimize its integration with standard care, and to fully exploit its therapeutic potential. Advances in protein engineering can further extend the utility of anti-GD2 mAbs, enabling the creation of radioimmunoconjugates, novel bispecific antibodies, and pre-targeting strategies, offering potent tumor selectivity while mitigating off-target toxicity.

P2, N=59, Completed, Memorial Sloan Kettering Cancer Center | Active, not recruiting --> Completed | Trial completion date: Jun 2026 --> Sep 2025 | Trial primary completion date: Jun 2026 --> Sep 2025

P2, N=46, Active, not recruiting, Memorial Sloan Kettering Cancer Center | Trial completion date: Jul 2025 --> Jul 2026 | Trial primary completion date: Jul 2025 --> Jul 2026

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.

Sensitivity analyses and unadjusted comparisons supported the results. In the indirect comparison, dinutuximab beta significantly extended PFS and increased ORR compared to naxitamab.

P2, N=93, Recruiting, Giselle Sholler | Trial completion date: Sep 2035 --> Sep 2036 | Trial primary completion date: Sep 2028 --> Sep 2029

In this case series, 4 patients were treated with the anti-GD2 monoclonal antibody naxitamab and granulocyte-macrophage colony-stimulating factor (GM-CSF) in combination with cyclophosphamide and topotecan between August 2021 and December 2022. This combined chemoimmunotherapy regimen was well tolerated in these heavily pretreated patients with R/R HR neuroblastoma who had few treatment options and overall poor prognoses.

P1, N=85, Completed, Memorial Sloan Kettering Cancer Center | Active, not recruiting --> Completed | Trial completion date: Jan 2026 --> Jul 2025 | Trial primary completion date: Jan 2026 --> Jul 2025