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
A Safety and Efficacy Study of hu14 in High-Risk Neuroblastoma Patients
Neuroblastoma is the most common type of solid cancer found outside the brain in young children. Generally, it affects children younger than 5 years old, with the average age when it is found being just 2 years. Most patients have 'high-risk' disease, with spread of the disease to different sites (metastases). This multinational study aims to find out how effective and safe the treatment of a monoclonal anti-GD2 antibody hu14.18K322A (daretabart) is when used together with chemotherapy to treat children and young people who have high-risk neuroblastoma.
Serial ctDNA and Molecular Residual Disease Monitoring in Neuroblastoma
This prospective observational study evaluates serial circulating tumor DNA (ctDNA) and molecular residual disease (MRD) monitoring in patients with neuroblastoma. The study aims to characterize baseline genomic alterations, assess ctDNA detectability and dynamic changes during treatment and follow-up, compare tumor-informed personalized MRD assays with fixed-panel assays, and determine the clinical utility of ctDNA/MRD for treatment response assessment, molecular remission evaluation, relapse surveillance, and early detection of disease progression.
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