Targeted therapies for leukaemia

medicine pills

Targeted therapies can stop cancer growing or spreading by targeting specific proteins and gene mutations that help the cancer to grow. 

Targeted therapies can be used alone or with other treatments like chemotherapy.
New drug treatments are being developed all the time. Some new drugs may be available to you as part of a clinical trial. Ask your consultant about this. 

Tell your doctor and your pharmacist about any other medications you are taking – both prescription and over-the-counter medications. Targeted therapies can interact with other drugs.

What are the side-effects?

These drugs have some mild side-effects. You may get side-effects after the first dose, a bit like an allergic reaction. Side-effects depend on the drugs used and include:

  • Nausea and diarrhoea
  • Fatigue
  • Leg aches
  • Muscle cramps
  • Swelling of fingers, eyelids, face or lower legs
  • Fever
  • Headaches
  • Rashes or itching
  • Chills
  • Breathing problems
  • Chest or tummy pain
  • Low blood pressure
  • Infections
  • Bleeding

Tell your nurse or doctor if you get any side-effects.


Types of targeted therapies for leukaemia

TKIs for Philadelphia-positive CML and ALL

Targeted therapy drugs called tyrosine kinase inhibitors (TKIs) can be used to treat leukaemia helped to grow by the abnormal Philadelphia chromosome. They are the main treatment for CML and are also used for Philadelphia-positive ALL.   

The Philadelphia chromosome causes your body to produce the enzyme tyrosine kinase, which makes the leukaemia cells grow and divide more quickly. TKIs work by blocking tyrosine kinase to slow the cancer’s growth. 

TKIs are normally taken as a tablet, once or twice a day, depending on the drug used. Examples of TKIs include imatinib (Glivec®) and dasatinib (Sprycel®). 

BTK inhibitors for CLL

BTK inhibitors block a protein called Bruton’s tyrosine kinase (BTK). BTK helps cancer cells to grow and survive.

By blocking the BTK, BTK inhibitors can slow the growth of CLL cells or destroy them. 

Examples of BTK inhibitors include Ibrutinib, Zanubrutinib, and Acalabrutinib. They are given as tablets.

You will stay on these drugs as long as they are working to control your CLL and you are not having any problems with the medication.

Possible side-effects of BTK inhibitors include increased risk of infection, due to a low white blood cell count, nausea and diarrhoea, pain in muscles and bones, bruising and bleeding, rash, fever, irregular heartbeat and tiredness (fatigue). 

BCL2 inhibitors for CLL

BCL2 inhibitors block a protein called BCL2. BCL2 interferes with the normal process where cells naturally die after a time. BCL2 helps CLL cells stay alive for longer, so they grow out of control. 

BCL2 inhibitors target the BCL2 protein so that the cancerous cells are not kept alive, instead they die off naturally. 

An example of a BCL2 inhibitor is Venetoclax. BCL2 inhibitors are given as tablets.

Possible side-effects of BCL2 inhibitors include increased risk of infection, due to a low white blood cell count, nausea and vomiting, constipation and diarrhoea, anaemia (low red blood cell count),
reduced appetite, headache and tiredness (fatigue). 

Monoclonal antibodies for CLL

Antibodies are proteins made naturally in your body that fight infection and cancer. Monoclonal antibodies are similar proteins made in the laboratory. Once in your body, they stick to specific
proteins on the surface of your white blood cells (lymphocytes),
including the abnormal ones. Your immune system attacks these
cells and kills them. Normal lymphocytes can then replace the
abnormal ones that have been destroyed. 

Examples of monoclonal antibodies used for people with CLL include rituximab, ofatumumab, alemtuzumab and obinutuzumab. 

Monoclonal antibodies are given into a vein by drip infusion. You will get your treatment in the hospital day ward. Usually you will have treatment for about 6 months.

Often monoclonal antibodies are given in combination with chemotherapy or other drugs. This is called combination therapy. 

Tips & Hints – taking TKIs

  • Take your medication exactly as your doctor recommends. Don’t stop taking your tablets, even if you no longer have any signs of disease.
  • If you find it hard to remember to take your tablets, plan ways to help you remember. For example, keep your pills somewhere where you will see them, set a reminder on your mobile phone or use a 7-day pill container. 
  • TKIs can cause muscle cramps - eating more calcium and magnesium-rich foods may help. Calcium is found in dairy products. Good sources of potassium include bananas and plain chocolate.
  • Avoid grapefruit, pomegranate and Seville oranges. Chemicals in these fruits can stop TKIs from working properly.
  • Ask your doctor about any other drugs and supplements that may stop your TKIs from working well or that may harm your health when you are taking TKIs. When you are prescribed any new medications ask your doctor or pharmacist to check whether they interact with your TKIs.
  • Look after your skin to try to avoid rashes. Moisturise your skin with a product recommended by your healthcare team and protect your skin from the sun. Avoid products that dry out or irritate the skin, such as soaps or perfumed products.
  • Use contraception if you are fertile and having sex. TKIs can damage an unborn baby.

I feel well. Why do I need medication?

If you stop taking your medication or miss even one or two doses you may relapse. This means your body will start to produce leukaemia cells again and you may develop symptoms. Keep taking your medication as prescribed, even if you are in remission and feel well.

For more information

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