Neuroblastoma Cancer Tumor in Children

What is Neuroblastoma Cancer?

Neuroblastoma is a type of rare childhood cancer tumor that develops from nerve cells in the fetus called neuroblasts. Typically, these cells mature and develop normally after birth, but occasionally, they can form cancerous tumors, causing neuroblastoma. 

Neuroblastoma tumors generally develop in the adrenal glands, which are located on top of the kidneys. However, they can also begin in or spread to other parts of the body, including the chest, neck, spine or spinal cord, abdomen, and bones. 

Neuroblastoma cancer is diagnosed in approximately 800 children in the United States each year. Ninety percent of cases occur in children under five years old, though the average age of diagnosis is between one and two years old. Still, because neuroblastomas form from fetal nerve cells, children as young as newborns can develop these cancer tumors, and neuroblastoma accounts for 50% of all cancer cases in infants.

Neuroblastoma Treatments and Prognosis

Neuroblastoma treatments for children vary based on age, stage, the type of cells involved in the cancer and whether there are additional copies of the MYCN gene present. The MYCN gene regulates cell growth. When mutated, it can cause cancerous tumors. Cancer tumors with the MYCN gene, like neuroblastoma, are more likely to spread and less likely to respond to treatment. Based on these factors, children are placed into different risk categories:

• Low Risk: Stage 1; Stage 2a and 2b in which more than 50% of the tumor was surgically removed and no additional copies of the MYCN gene
• Intermediate Risk:  Stages 2a and 2b in which less than 50% of the tumor was removed and no additional copies of the MYCN gene; Stage 3 with no additional copies of the MYCN gene; Stage 4 and under 1 year of age with no additional copies of the MYCN gene; Stage 4S with no additional copies of the MYCN gene
• High Risk: Stages 2a, 2b and 3 with extra copies of the MYCN gene; Stage 4 and 4S with extra copies of the MYCN gene

Treatment plans are determined by risk category and typically consist of a combination of surgery to remove as much of the tumor as possible, chemotherapy and radiation therapy. Stage 4 neuroblastoma and higher risk patients often receive additional treatments, such as immunotherapy, bone marrow transplants, or newer treatments like MIBG therapy, which uses radioactive MIBG to target neuroblastoma cells and release radiation.1

Risk categories also determine the five-year survival rates for children with neuroblastoma. 

For low-risk patients: about 95%

For intermediate-risk patients: 80-90%

For high-risk patients: 40-50%

Approximately two-thirds of all children diagnosed with neuroblastoma cancer will have some metastatic disease, and infants have a better chance than older children of remaining free of neuroblastoma after treatment.

Solving Kids’ Cancer, alongside Children’s Neuroblastoma Cancer Foundation and Solving Kids’ Cancer UK, recently held the inaugural Neuroblastoma Parent Global Symposium. This virtual event brought together families affected by neuroblastoma from around the world with those who work to treat, help and support them. During the event, international experts shared information on treatment and clinical trials for neuroblastoma treatments through live presentations, panel sessions and Q&As.

Solving Kids’ Cancer’s Role in Neuroblastoma Research

Despite multi-modal treatment with chemotherapy, surgery and radiation, children with high-risk neuroblastoma have a more difficult prognosis. A recent combination of chemotherapy and anti-GD2 treatment has shown promise for some children with neuroblastoma who have relapsed or refractory disease.2

In our research approach, Solving Kids’ Cancer is championing clinical studies using novel combinations of new modalities like immunotherapy and targeted therapies against validated biomarkers along with developing better drug delivery and identifying new targets for immunotherapy and targeted agents. Building on this, Solving Kids’ Cancer is working in tandem with the research community to move these promising new modalities much earlier in the course of treatment for newly diagnosed children.

These collective efforts hold great promise for improved outcomes and are enabled by our ability to remain independent from single institutions and work with collaborators internationally.

Solving Kids’ Cancer is currently supporting seven neuroblastoma clinical trial and development projects, in addition to the 17 previously supported. A recent achievement was spearheading an international effort to bring Lorlatinib, an investigational drug candidate in development for lung cancer, to a transatlantic new clinical trial for kids with neuroblastoma. While the trial has not yet concluded, the early results for Kira, a teenager in Scotland, are very promising.

Solving Kids’ Cancer believes the launch and support of just one clinical trial can save lives and change the world for children with cancer. Your gift makes innovative clinical trials for kids like Kira who are fighting neuroblastoma possible — because every kid deserves to grow up®.