New Treatments & Clinical Trials for Neuroblastoma

Neuroblastoma is a cancer of immature nerve cells that mostly affects children under five. It accounts for about 15% of childhood cancer deaths. Treatment depends on risk group — low-risk tumors may need no treatment at all, while high-risk cases require surgery, chemotherapy, stem cell transplant, immunotherapy, and radiation.

What's actually going on in research

Trials are testing new immunotherapy combinations, targeted drugs that exploit neuroblastoma's genetic vulnerabilities, cellular therapies including CAR T-cells and natural killer cells, and ways to make existing treatments less toxic. Research focuses heavily on high-risk disease and on preventing relapse, which remains common and often fatal.

What to know before you search

Eligibility depends on risk group, whether the tumor is newly diagnosed or relapsed, prior treatments received, and specific genetic features of the tumor like MYCN amplification or ALK mutations.

What types of trials are currently open

• Treatment trials — Testing new drug combinations, immunotherapies, or targeted drugs to improve cure rates for high-risk neuroblastoma or reduce treatment intensity for lower-risk disease.
• Relapse trials — Testing treatments for neuroblastoma that has come back after initial therapy, including novel immunotherapies and cellular therapies.
• Stem cell transplant trials — Studies comparing different transplant approaches or testing whether some children can skip transplant.
• Supportive care trials — Testing ways to reduce pain, neuropathy, hearing loss, and other side effects of neuroblastoma treatment.
• Observational studies — Following children with neuroblastoma to understand how tumors behave, which genetic features predict outcomes, and long-term effects of treatment.