Immunotherapy for Neuroblastoma

Immunotherapy is the use of medicines to help a patient’s own immune system recognize and destroy cancer cells more effectively. Several types of immunotherapy are now being studied for use against neuroblastoma (some of which are described in What’s New in Neuroblastoma Research?), and some are now being used to treat it.

Anti-GD2 monoclonal antibodies

Monoclonal antibodies are lab-made versions of immune system proteins that can attach to a very 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 (Unituxin)

This monoclonal antibody is typically given together with cytokines (immune system hormones) such as GM-CSF and interleukin-2 (IL-2), as well as isotretinoin, to help the body’s immune system recognize and destroy neuroblastoma cells. It is typically used as part of the treatment for children with high-risk neuroblastoma, following a stem cell transplant.

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.

Naxitamab (Danyelza)

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 one 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
  • Rapid heartbeat
  • Fever
  • Vomiting
  • Cough
  • Nausea
  • Diarrhea
  • Low 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.

More information about immunotherapy

To learn more about how drugs that work on the immune system are used to treat cancer, see Cancer Immunotherapy.

To learn about some of the side effects listed here and how to manage them, see Managing Cancer-related Side Effects.

The American Cancer Society medical and editorial content team

Our team is made up of doctors and oncology certified nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

Dome JS, Rodriguez-Galindo C, Spunt SL, Santana VM. Chapter 92: Pediatric solid tumors. In: Neiderhuber JE, Armitage JO, Doroshow JH, Kastan MB, Tepper JE, eds. Abeloff’s Clinical Oncology. 6th ed. Philadelphia, PA. Elsevier; 2020.

National Cancer Institute. Neuroblastoma Treatment (PDQ). 2020. Accessed at https://www.cancer.gov/types/neuroblastoma/hp/neuroblastoma-treatment-pdq on April 9, 2021.

Park JR, Hogarty MD, Bagatell R, et al. Chapter 23: Neuroblastoma. In: Blaney SM, Adamson PC, Helman LJ, eds. Pizzo and Poplack’s Principles and Practice of Pediatric Oncology. 8th ed. Philadelphia Pa: Lippincott Williams & Wilkins; 2021.

Shohet JM, Lowas SR, Nuchtern JG. Treatment and prognosis of neuroblastoma. UpToDate. 2021. Accessed at https://www.uptodate.com/contents/treatment-and-prognosis-of-neuroblastoma on April 9, 2021.

Pinto NR, Applebaum MA, Volchenboum SL, et al. Advances in risk classification and treatment strategies for neuroblastoma. J Clin Oncol. 2015: 30;3008-3017.

Yu AL, Gilman AL, Ozkaynak MF, et al. Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. N Engl J Med. 2010;363:1324–1334.

References

Dome JS, Rodriguez-Galindo C, Spunt SL, Santana VM. Chapter 92: Pediatric solid tumors. In: Neiderhuber JE, Armitage JO, Doroshow JH, Kastan MB, Tepper JE, eds. Abeloff’s Clinical Oncology. 6th ed. Philadelphia, PA. Elsevier; 2020.

National Cancer Institute. Neuroblastoma Treatment (PDQ). 2020. Accessed at https://www.cancer.gov/types/neuroblastoma/hp/neuroblastoma-treatment-pdq on April 9, 2021.

Park JR, Hogarty MD, Bagatell R, et al. Chapter 23: Neuroblastoma. In: Blaney SM, Adamson PC, Helman LJ, eds. Pizzo and Poplack’s Principles and Practice of Pediatric Oncology. 8th ed. Philadelphia Pa: Lippincott Williams & Wilkins; 2021.

Shohet JM, Lowas SR, Nuchtern JG. Treatment and prognosis of neuroblastoma. UpToDate. 2021. Accessed at https://www.uptodate.com/contents/treatment-and-prognosis-of-neuroblastoma on April 9, 2021.

Pinto NR, Applebaum MA, Volchenboum SL, et al. Advances in risk classification and treatment strategies for neuroblastoma. J Clin Oncol. 2015: 30;3008-3017.

Yu AL, Gilman AL, Ozkaynak MF, et al. Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. N Engl J Med. 2010;363:1324–1334.

Last Revised: April 28, 2021

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