Neuroblastoma and other peripheral nervous cell tumors

• 1964Melphalan approved (FDA)
• 1994Iobenguane approved (FDA)
• 2015Dinutuximab approved (FDA)
• 2017Dinutuximab beta approved (FDA)
• 2020Naxitamab approved (FDA)

Trials started per year, stacked by phase (darker = later phase). Source: ClinicalTrials.gov via Clin2, which tracks studies recruiting in recent years — early years undercount.

Neuroblastoma is not preventable, but it is treatable, and for some children it is curable. Low- and intermediate-risk disease often responds well to surgery and chemotherapy, with high cure rates. High-risk disease remains harder to treat, yet intensive therapy combined with newer immunotherapy drugs such as dinutuximab and naxitamab has brought long-term remission to a meaningful subset of patients. Treatment has advanced steadily since the first chemotherapy agents were approved in the 1960s, and the pace has quickened with three immunotherapy approvals since 2015. More than a hundred clinical trials are now underway, including several late-stage studies, so the trajectory is toward more children surviving. Still, not every child can access these treatments, and cure is not yet possible for most patients overall.

What does it mean that neuroblastoma is curable in some children?

It means certain risk groups, especially low- and intermediate-risk disease, can often be cured with surgery and chemotherapy. High-risk disease is harder to treat, but some children achieve long-term remission with intensive therapy and newer immunotherapies.

How has treatment improved over time?

The first chemotherapy for this disease was approved in 1964. Since then, targeted imaging and therapy agents arrived in the 1990s, and three immunotherapy drugs were approved between 2015 and 2020, giving more options for high-risk patients.

Are there new treatments being tested?

Yes. More than 100 clinical trials have started in the last five years, including seven phase-3 studies. Around 140 trials are currently active, testing new combinations and approaches to improve survival and reduce side effects.

Is the survival rate improving?

Overall, survival has improved substantially over recent decades, especially for children with low- and intermediate-risk disease. For high-risk disease, newer immunotherapies have increased the chance of long-term remission, though it remains a difficult diagnosis.

Can every child get these newer treatments?

No. Availability depends on the child's risk group, the treatment center, and regional access to specialized care. Immunotherapies like dinutuximab and naxitamab are not universally available, and they are typically part of intensive treatment protocols.