Neuroblastoma is the most common solid tumor in infants and one of the most common pediatric cancers overall, arising from immature nerve cells most often in the adrenal glands. Low-risk cases often resolve on their own, while high-risk neuroblastoma has a devastating prognosis despite aggressive treatment.
What's actually going on in research
Dinutuximab, an antibody targeting GD2 on neuroblastoma cells, is now part of standard high-risk treatment and is being combined with other immunotherapies in trials. DFMO (difluoromethylornithine), which blocks an enzyme needed for neuroblastoma growth, is in trials as maintenance therapy to prevent relapse. ALK inhibitors are showing benefit in the 7–10% of neuroblastomas with ALK mutations, and trials are expanding their use.
Dinutuximab combinations
The GD2-targeting antibody dinutuximab is combined with immunostimulatory cytokines; trials are adding checkpoint inhibitors and CAR-T cells targeting GD2 to further improve responses.
DFMO maintenance
DFMO blocks polyamine synthesis that neuroblastoma cells need to grow, and trials are testing it as prolonged maintenance therapy to keep high-risk disease in remission.
ALK inhibitor therapy
Crizotinib and lorlatinib target ALK-mutated neuroblastomas, and trials are testing these drugs in the front-line setting for patients whose tumors carry ALK amplifications or mutations.
What to know before you search
Eligibility depends on neuroblastoma risk group (low, intermediate, high), MYCN amplification, ALK mutation status, and stage of disease.
What types of trials are currently open
- High-risk neuroblastoma trials — Testing new drug combinations, immunotherapy, and maintenance strategies for stage 4 neuroblastoma.
- Immunotherapy trials — Evaluating dinutuximab combinations, CAR-T targeting GD2, and checkpoint inhibitors.
- ALK-targeted trials — Testing ALK inhibitors in ALK-mutated neuroblastoma in front-line and relapsed settings.
- Maintenance therapy trials — Evaluating DFMO and other agents to prevent relapse after completion of front-line therapy.
- Low-risk observation trials — Testing observation versus minimal treatment in low-risk neuroblastoma to avoid over-treatment.
Recently added Neuroblastoma trials
Haploidentical Donor Cytokine-Induced Memory-Like Natural Killer Cells (CIML-NK) for Relapsed & Refractory Neuroblastoma
The goal of this study is to demonstrate that cytokine-induced memory-like natural killer cells (CIML-NK cells) can be generated from donor cells and infused safely into patients with relapsed or refractory neuroblastoma during dinutuximab-based therapy.
Phase I Study of Becotatug Vedotin for Safety and Efficacy in EGFR-Positive Pediatric Relapsed/Refractory or Metastatic Solid Tumors
There is a significant unmet medical need for effective therapies for pediatric relapsed/refractory solid tumors. EGFR is highly and stably expressed in multiple pediatric solid tumor subtypes, and adult Phase I data of Becotatug Vedotin demonstrated a manageable safety profile and promising antitumor activity in EGFR-positive advanced solid tumors.This is a multicenter, non-randomized, single-arm, open-label Phase I clinical trial sponsored by Sun Yat-sen University Cancer Center (SYSUCC). The trial evaluates the safety, tolerability, pharmacokinetics (PK), immunogenicity, and preliminary efficacy of Becotatug Vedotin-an EGFR-targeted antibody-drug conjugate (ADC)-in pediatric patients with EGFR-positive relapsed/refractory or metastatic solid tumors.
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