Immunotherapy for Neuroblastoma
Immunotherapy is the use of medicines to help a patient’s own immune system recognize and destroy cancer cells more effectively.
Anti-GD2 monoclonal antibodies
Monoclonal antibodies are lab-made versions of immune system proteins that can attach to a specific target on cells in the body. These antibodies can be injected into the blood to seek out and attach to cancer cells.
Many neuroblastoma cells have large amounts of a substance called GD2 on their surfaces. Monoclonal antibodies that attach to GD2 can help the body’s immune system find and destroy these cancer cells.
Dinutuximab can be used to treat children with high-risk neuroblastoma. This monoclonal antibody is used with a drug that triggers the bone marrow to make more immune cells, called GM-CSF, and isotretinoin to help the body’s immune system recognize and destroy neuroblastoma cells. It is currently part of treatment after receiving a stem cell transplant.
New clinical trials are looking at whether giving immunotherapy before transplant is better.
This drug is given as an infusion into a vein (IV) over many hours, for 4 days in a row. This is done about once a month, usually for a total of about 5 cycles of treatment. Other medicines are given before and during each infusion to help with possible side effects such as pain or infusion reactions.
Possible side effects
Dinutuximab can cause side effects, some of which can be serious. Possible side effects include:
- Nerve pain (which can sometimes be severe)
- Leaking of fluid from small blood vessels (which can lead to low blood pressure, fast heart rate, shortness of breath, and swelling)
- Infusion reactions (which can lead to airway swelling, trouble breathing, and low blood pressure)
- Eye and vision problems
- Fever
- Vomiting
- Diarrhea
- Itching
- Trouble urinating
- Infections
- Low blood cell counts
- Changes in mineral levels in the blood
Other side effects are possible as well. Talk to your child's treatment team to learn more about the possible side effects and what can be done about them.
This monoclonal antibody is given together with the cytokine (immune system hormone) GM-CSF to help the body’s immune system recognize and destroy neuroblastoma cells.
Naxitamab can be used in patients who are at least 1 year old and who have high-risk neuroblastoma that is in their bones or bone marrow and that has come back or started to grow again after initially responding to treatment.
This drug is given as an infusion into a vein (IV) over 30 to 60 minutes on days 1, 3, and 5 of each 4-week cycle. Other medicines are given before and during each infusion to help with possible side effects such as pain or infusion reactions.
Possible side effects
Naxitamab can cause side effects, some of which can be serious. Possible side effects include:
- Nerve pain (which can sometimes be severe)
- Infusion reactions (which can lead to airway swelling, trouble breathing, and low blood pressure)
- Eye and vision problems
- Fast heartbeat
- Fever
- Vomiting
- Cough
- Nausea
- Diarrhea
- Low or high blood pressure
- Itching
- Trouble urinating
- Infections
- Low blood cell counts
- Changes in mineral levels in the blood
Other side effects are possible as well. Talk to your child's treatment team to learn more about the possible side effects and what can be done about them.
Vaccines
Several cancer vaccines are also being studied for use against neuroblastoma. For these vaccines, injections of modified neuroblastoma cells or other substances are given to train the child’s own immune system to attack cancer cells.
CAR T-cell therapies
CAR T-cell therapy is a promising new way to get a patient's own immune cells, called T cells, to fight cancer by changing them in the lab so they can find and destroy cancer cells. The T cells used in CAR T-cell therapies are removed from the blood and changed in the lab by adding a gene for a lab-made receptor (called a chimeric antigen receptor or CAR), which helps them attack specific cancer cells.
Studies are now testing if CAR T cells that target GD2 (or other substances) on neuroblastoma cells can be helpful in treating this disease. Most of these are clinical trials that are ongoing or in the planning phase.
Several other types of immunotherapy are also being studied for use against targets found on neuroblastoma cells. Many of these are in clinical trials. If you have questions about these or other investigational treatments for neuroblastoma, talk to your child’s cancer care team.
More information about immunotherapy
To learn more about how drugs that work on the immune system are used to treat cancer, see Cancer Immunotherapy.
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- References
Developed by the American Cancer Society medical and editorial content team with medical review and contribution by the American Society of Clinical Oncology (ASCO).
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Last Revised: June 26, 2025
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