Introduction

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:

• surgery - to remove the tumour
• chemotherapy – medicine that kills cancer cells
• radiotherapy – high doses of radiation that kill cancer cells.

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

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.

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.

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.