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Retinoids and immunotherapy

Retinoids and immunotherapy drugs are sometimes used to treat high-risk or recurrent neuroblastoma. They are given after high-dose chemotherapy and stem cell transplant, and may be called maintenance therapy. Retinoids and immunotherapy drugs are used to:

  • destroy cancer cells left behind and lower the risk that the cancer will recur (called adjuvant therapy)
  • strengthen the immune system

There are 4 drugs commonly used together to treat neuroblastoma: a retinoid and 3 immunotherapy drugs. Your child’s healthcare team will consider your child’s personal needs to plan the drugs, doses and schedules of retinoids and immunotherapy. Your child may also receive other treatments.


Retinoids are a form of vitamin A. They help some cancer cells mature, or differentiate, into normal cells. As a result, retinoids are also called differentiating agents.

The retinoid used for high-risk or recurrent neuroblastoma is called 13-cis-retinoic acid (Isotretinoin, Accutane). It is given by mouth for 6 months following stem cell transplant.


Immunotherapy is a type of biological therapy that uses natural or artificial substances that change the way cells behave. Different types of immunotherapies work in different ways. Some types kill, control or change how cancer cells behave. Other types strengthen the body’s immune system, control symptoms or lessen side effects of treatment.

Interleukins are a group of hormone-like substances made by white blood cells. They stimulate the immune system to recognize and destroy cancer cells. Interleukin-2 (Aldesleukin, Proleukin) is the drug used for high-risk or recurrent neuroblastoma. It may be given by injection into a vein (called intravenous, or IV, injection) or under the skin (called subcutaneous injection).

Granulocyte growth factors may be used to lessen some of the side effects of cancer treatments. They stimulate the bone marrow to make more white blood cells. This helps reduce the risk of infection after high-dose chemotherapy and stem cell transplant. Sargramostim (Leukine) is the drug used. It is given by an injection under the skin (called subcutaneous injection).

Monoclonal antibodies are a type of targeted immunotherapy used to treat neuroblastoma. Dinutuximab (Unituxin) is a monoclonal antibody that targets a protein called GD2, which neuroblastoma cells express. It works by attaching to neuroblastoma cells that have the GD2 protein and then stimulating the body’s immune system to attack and kill the neuroblastoma cells. It is usually given along with the immunotherapies above.

Side effects

Side effects can happen with any type of treatment for neuroblastoma, but every child’s experience is different. Some children have many side effects. Others have few or none at all.

Side effects can develop any time during, immediately after or a few days or weeks after therapy. Sometimes late side effects develop months or years after therapy. Most side effects go away on their own or can be treated, but some side effects may last a long time or become permanent.

Side effects of retinoids and immunotherapy will depend mainly on the type and dose of drug or drugs used and the child’s overall health.

Some common side effects of retinoids include:

Some common side effects of immunotherapy include:

Other side effects can develop months or years after treatment for neuroblastoma. Find out more about late effects for neuroblastoma.

Tell your child’s healthcare team if your child has these side effects or others you think might be from biological therapy. The sooner you tell them of any problems, the sooner they can suggest ways to help your child deal with them.

Information about specific cancer drugs

Details on specific drugs change quite regularly. Find out more about sources of drug information and where to get details on specific drugs.

Questions to ask about therapy

Find out more about immunotherapy and targeted therapy, such as targeted immunotherapy. To make the decisions that are right for you, ask your healthcare team questions about immunotherapy and questions about targeted therapy.

monoclonal antibody

A substance that can find and bind to a particular target molecule (antigen) on a cancer cell.

Monoclonal antibodies can interfere with a cell’s function or can be used to carry drugs, toxins or radioactive material directly to a tumour.


Dr Robert Day Targeting an “evil twin” enzyme in prostate cancer

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