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

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.

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

The patient was treated with systemic conventional and high-dose chemotherapy combined with intrathecal Topotecan. This case suggests that anti-GD2 immunotherapy, used as consolidation treatment, may improve the prognosis of patients with advanced retinoblastoma and potentially reduce the toxicity associated with standard therapies. Further clinical investigation is warranted to validate these findings.

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

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

P1, N=44, Active, not recruiting, University Hospital Southampton NHS Foundation Trust | Trial completion date: Jul 2025 --> Nov 2025

Several proteins not previously associated, or only indirectly linked, with beta cell function, apoptosis, or proliferation were identified and characterized for the first time in insulinoma. These findings provide new insights and potential targets for the treatment of pancreatic cancer.

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