Understanding Neuroblastoma: A Guide for Families
Neuroblastoma is a rare childhood cancer that starts in very early forms of nerve cells called neuroblasts. While it is the most common solid tumor found outside the brain in children, its behavior is often unpredictable. Some tumors can be aggressive, growing and spreading quickly, while others grow slowly. In some remarkable cases, particularly in infants, the cancer can even disappear on its own through a process called spontaneous regression.
This cancer develops from the sympathetic nervous system, which controls essential body functions like heart rate and blood pressure. Because these nerve cells are located throughout the body, tumors can appear in several places, most commonly in the adrenal glands on top of the kidneys. They can also form in the abdomen, chest, neck, or pelvis. Neuroblastoma is almost exclusively a disease of early childhood, with over 90% of cases diagnosed in children under the age of five.
How is Neuroblastoma Diagnosed and Classified?
Once neuroblastoma is suspected, doctors perform a series of tests to confirm the diagnosis and gather critical information about the tumor. This process helps them classify the disease into a risk group—low, intermediate, or high. This risk classification is the single most important factor in determining the best treatment plan for a child.
Age at Diagnosis
A child's age is a key predictor of outcome. Infants diagnosed under 18 months old often have a much better prognosis, even if the cancer has spread. Some infants with a specific stage of disease (Stage MS), where the cancer has only spread to the skin, liver, or bone marrow, have an excellent chance of recovery, sometimes with very little treatment.
Tumor Stage
The stage of the cancer describes how far it has spread from its original location. Doctors use imaging scans to determine if a tumor is localized (contained in one area) or metastatic (spread to distant parts of the body). A localized tumor that can be surgically removed is associated with a more favorable outlook. In contrast, cancer that has spread to distant sites like the bones or bone marrow (Stage M) is considered higher risk and requires more intensive therapy.
Tumor Biology
Beyond location, the genetic makeup of the cancer cells provides crucial clues about how the tumor will behave. One of the most important genetic markers is the MYCN gene. If a tumor has extra copies of this gene (a condition called MYCN amplification), it signals that the cancer is likely to be very aggressive. A child with a MYCN-amplified tumor is automatically placed in the high-risk category, regardless of their age or stage, and will receive the most intensive treatment available.
Navigating Treatment Options
The treatment for neuroblastoma is tailored to each child's specific risk group. A dedicated healthcare team works closely with the family to develop a plan that may involve one or more of the following therapies.
Surgery
For many children with low-risk, localized tumors, surgery to remove the cancer may be the only treatment needed. The primary goal is to remove as much of the tumor as possible without harming nearby organs. In higher-risk cases, chemotherapy may be given first to shrink the tumor, making the subsequent surgery safer and more effective.
Chemotherapy
Chemotherapy uses powerful drugs to kill cancer cells throughout the body. For intermediate-risk disease, it is often used before surgery to help ensure the entire tumor can be removed. In high-risk neuroblastoma, chemotherapy is a cornerstone of treatment, used to attack both the primary tumor and any cancer cells that have spread elsewhere. These medicines are typically administered in cycles to give the body time to rest and recover.
Radiation Therapy
This treatment uses high-energy beams to destroy cancer cells that may remain after surgery and chemotherapy. Radiation is most often used for high-risk neuroblastoma to treat the original tumor site, reducing the chance of the cancer returning in that area. The therapy is carefully planned to target cancer cells while sparing surrounding healthy tissue as much as possible.
Immunotherapy and Stem Cell Transplant
These intensive treatments are reserved for children with high-risk neuroblastoma. An autologous stem cell transplant allows doctors to use very high doses of chemotherapy to eliminate the cancer. Before this treatment, the child’s own blood-forming stem cells are collected and stored. After the high-dose chemotherapy, these cells are returned to the body to rebuild the bone marrow. Immunotherapy is then often used to help the child's own immune system find and destroy any remaining cancer cells.
A New Era in Treatment
Research is rapidly changing the future of neuroblastoma care, leading to smarter, more effective, and gentler therapies. This era of precision medicine offers new hope by tailoring treatments to the unique biology of each child’s tumor.
Targeted Drug Therapy
Instead of treatments that affect all fast-growing cells, targeted drugs focus on specific weaknesses within the cancer cells themselves. For example, the drug lorlatinib has shown significant promise for children whose tumors are driven by a mutation in the ALK gene. This drug, when combined with chemotherapy, has successfully shrunk tumors in many patients and is now being studied as a frontline treatment for newly diagnosed children with this specific genetic marker.
Harnessing the Immune System
Immunotherapy empowers a child’s own immune system to fight cancer. One revolutionary approach is CAR T-cell therapy. In this treatment, a child’s immune cells (T-cells) are collected and re-engineered in a lab to recognize a protein called GD2, which is found on neuroblastoma cells. These supercharged cells are then returned to the body to hunt down and destroy the cancer. Early trials have shown remarkable success in children who had exhausted other options.
Cutting Off the Tumor's Lifeline
Tumors require a blood supply to grow and survive. Anti-angiogenic drugs work by cutting off this supply line. The drug bevacizumab, for example, prevents tumors from forming the new blood vessels they need to expand. Clinical trials have shown that adding bevacizumab to standard chemotherapy can improve treatment response rates for some children with high-risk neuroblastoma.
Long-Term Health for Survivors
Thanks to modern treatments, a majority of children with neuroblastoma survive into adulthood. This success has shifted focus toward managing the long-term health of these survivors. The powerful therapies that save lives can sometimes cause late effects, which are health issues that appear months or years after treatment ends. Every survivor receives a personalized Survivorship Care Plan to guide their ongoing health monitoring.
Hearing Loss
Certain chemotherapy drugs can be toxic to the delicate structures of the inner ear, potentially leading to hearing loss. This effect may not be apparent until after treatment and can range from minor to significant. Regular hearing tests with an audiologist are a critical part of long-term care for any child who received these medications.
Growth and Bone Health
Cancer treatment can interfere with a child’s natural growth. Radiation therapy, if directed at areas with growing bones, can sometimes lead to issues like scoliosis (a curved spine) or uneven limb length. Both chemotherapy and radiation can also affect the endocrine system, particularly the thyroid gland, which regulates growth. Regular monitoring of height, development, and hormone levels is essential to ensure long-term well-being.
Risk of Second Cancers
Although uncommon, the same therapies that cure neuroblastoma can slightly increase the risk of developing a different cancer later in life. Both radiation and some chemotherapy drugs work by damaging the DNA of cancer cells, but this can occasionally affect healthy cells as well. This is a key reason why lifelong follow-up is so important, as it allows the healthcare team to monitor for any new problems and address them at the earliest possible stage.
