Targeted therapy for neuroblastoma

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Targeted therapy uses drugs to target specific molecules (such as proteins) on or inside cancer cells to stop the growth and spread of cancer and limit harm to normal cells. Targeted therapy is sometimes used to treat neuroblastoma that has the ALK mutation. The anaplastic lymphoma kinase (ALK) gene sends signals to proteins that make cells grow and divide. When a cancer has an ALK mutation, drugs called ALK inhibitors can be used to treat it. ALK inhibitors, such as crizotinib, block the signals that tell the cancer cells to divide, so the cancer cells stop growing. Targeted therapy may also be called molecular targeted therapy.

Your child may have targeted therapy to treat neuroblastoma that no longer responds to other treatments or comes back (recurs) after treatment, or as part of initial treatment for high-risk neuroblastoma.

The healthcare team will consider your child's personal needs to plan the drugs, doses and schedules of targeted therapy. Your child may also receive other treatments.

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. Other children have only a few side effects.

Targeted therapy attacks cancer cells but doesn't usually damage healthy cells, so there are usually fewer and less severe side effects than with chemotherapy or radiation therapy. Chemotherapy and radiation therapy can damage healthy cells along with cancer cells.

If your child develops side effects, they can happen any time during, immediately after or a few days or weeks after targeted therapy. Sometimes late side effects develop months or years after targeted 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 targeted therapy will depend mainly on the type of drug or combination of drugs, the dose, how the drugs are given and your child's overall health. Some common side effects of targeted therapy for neuroblastoma are:

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

Tell the healthcare team if your child has these side effects or others you think might be from targeted 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 targeted therapy

Find out more about targeted therapy. To make the decisions that are right for your child, ask the healthcare team questions about targeted therapy.

Expert review and references

  • Meredith Irwin, MD
  • Daniel Morgenstern, MD