Targeted therapy for AML

Targeted therapy uses drugs that attack specific features of leukaemia cells to stop them growing and spreading.

Learn more about:

• Overview
• Side effects of targeted therapy
• Other targeted therapy drugs
• Video: What are drug therapies?

Overview

Venetoclax is a type of targeted therapy drug that may be used for people with certain subtypes of AML and those who are not able to have high-dose chemotherapy.

Venetoclax blocks the action of BCL-2, a protein that helps leukaemia cells survive. It is taken as a tablet in combination with a chemotherapy drug called azacitidine. Some people may need to stay in hospital for the first 3–4 days of treatment while the dose is adjusted. Your doctor will advise you to drink plenty of water during the first few weeks of treatment. This helps to prevent tumour lysis syndrome (TLS). TLS may occur when leukaemia cells die and release a chemical called uric acid. A build-up of uric acid can damage the kidneys.

Side effects

Other possible side effects include:

• increased risk of infection
• breathing problems
• fatigue (tiredness)
• diarrhoea
• nausea and/or vomiting.

Speak to your treatment team if you experience any side effects, as they can often be treated with medicines.

Other targeted therapy drugs

AML may be treated with other targeted therapy drugs such as midostaurin and gemtuzumab ozogamicin (which are both used in combination with chemotherapy). People with relapsed AML may be offered a drug called gilteritinib.

For more on this, see Targeted therapy and listen to our podcast on New Cancer Treatments.

If you have cancer, drug therapy may play a big role in your treatment plan. Watch this short video to learn more about drug therapies, including targeted and immunotherapy.

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