Targeted therapy factsheet


Targeted therapy is a type of cancer treatment. It uses special medications that target a specific part of the cancer cells to stop them from growing and spreading. 

Cancer cells have changed or mutated as they grow and split into new cells. Genes are the instructions for how your body grows and works. They are found inside cells and are copied every time a cell splits and grows.

When a cell changes or mutates, the genes inside will also change or mutate. Changes in genes can build up over time and cause tumours and cancer to grow. 

There are many different types of cancer and many different things that cause them. Some cancers will have a specific feature on their surface or inside their cells. 

The medicine used in targeted therapy is specially made to target specific features in the cancer cells.

Targeted therapy can be used alone or with other treatments like:

Targeted therapy generally has fewer side effects, but it will only work on certain types of cancers that have specific features in their cells.

Preparing for treatment

Using targeted therapy will depend on:

  • the specific cancer being treated
  • the types of symptoms
  • your child's general health outside of the cancer.

Your child will have tests to figure out: 

  • what type of cancer they have 
  • whether targeted therapy can be used
  • the best-targeted therapy medicine to use. 

Tests can include:

  • blood tests
  • biopsy – where a sample of the tumour is taken and tested.

During treatment

Targeted therapy is different for every child.

Targeted therapy medicine can be given:  

  • as tablets or capsules that are swallowed
  • through an intravenous (IV) cannula – a small plastic tube inserted into a vein in the arm
  • through a central venous catheter (CVC) or port – a longer plastic tube inserted into a vein under the arm or in the chest, ending near the heart
  • as an injection under the skin.

It is common for children to react to their first dose of targeted therapy medicine, so the first dose is given slowly.

Your child’s treatment team will check how your child is doing during treatment. They can give your child other types of medicine to help prevent side effects like nausea and vomiting.

Let your child’s doctor know if you have any questions about treatment. They will let you know if there are any special instructions after each dose.

Length of treatment

How long your child continues targeted therapy depends on: 

  • the aim of the treatment
  • how the cancer responds to treatment 
  • any side effects your child has.

Medications can be given:

  • in cycles with rest periods in between
  • every day without breaks for a certain period
  • every day for several months or years.


Side effects from targeted therapy are generally milder than those from other treatments.

They will usually go away after treatment has finished.

Common side effects include:

  • eye problems  
  • loss of hair and hair colour  
  • changes to skin and nail texture
  • feeling very tired 
  • mouth sores
  • high blood pressure 
  • problems with blood clotting or wound healing 

Your child’s treatment team can help manage some side effects with medication, fluids, and rest.

After treatment

Children with cancer will have regular checkups with their doctors after treatment is finished.

This is to monitor:

  • how your child is recovering from treatment
  • whether the chemotherapy worked
  • whether any further treatment is needed.

If targeted therapy is working, the cancer should start to shrink or go away completely.

In some cases, the cancer will be stable. This means it does not grow but also does not shrink or disappear.

Children with stable cancer can often live for many years and have a good quality of life.

After the first few years, follow-up appointments will change to monitoring:

  • growth
  • development 
  • long-term or late side effects from treatment. 


Limitations of targeted therapy

Targeted therapy may not be the best treatment for every person with cancer. 

It is important to know that: 

  • targeted therapy will not work if your child’s cancer does not have the specific features the medicine targets 
  • sometimes, the cancer does not respond to the medicine, even if it has specific features
  • the response to the treatment may not last forever.  

When to see your doctor

Your child's treatment team will give you contact information for any questions or concerns about your child's health while at home during treatment. 

If your child has any of the following symptoms during treatment, call Triple Zero (000) for an ambulance or go to the emergency department:

  • difficulty breathing 
  • convulsions or seizures
  • signs of infection
  • fever
  • looking extremely unwell. 

Support for families

A cancer diagnosis can leave families and children feeling overwhelmed, scared, anxious, and upset. Practical and emotional support during and after treatment is essential and can come from: 

  • family
  • friends
  • healthcare professionals
  • specialised support services.

Speak to your child’s treatment team for information about support services.

Last updated Monday 25th March 2024


This factsheet is provided for general information only. It does not constitute health advice and should not be used to diagnose or treat any health condition.

Please consult with your doctor or other health professional to make sure this information is right for you and/or your child.

The Sydney Children’s Hospitals Network does not accept responsibility for inaccuracies or omissions, the interpretation of the information, or for success or appropriateness of any treatment described in the factsheet.

© Sydney Children’s Hospitals Network 2024