Targeted therapy for acute lymphoblastic leukaemia

Targeted therapies are drugs designed to identify and attack cancer cells, while doing as little damage as possible to normal cells. They work by ‘targeting’ specific proteins on or in the leukaemia cells.

They may be used in combination with other treatments such as chemotherapy.

Tyrosine Kinase Inhibitors (TKI)

Tyrosine kinase inhibitors (TKIs) are used to treat Ph+ ALL. Tyrosine kinase is a protein made by cells. It sends signals to cells that regulate their growth. In Ph+ALL all the leukaemia cells have an abnormal gene called BCR-ABL. It makes the cells produce too much tyrosine kinase and this causes changes in the blood and bone marrow.

Tyrosine kinase inhibitors work by blocking (inhibiting) signals within leukaemia cells. This makes the cells die. The most commonly used TKI is imatinib. You take it as a tablet. Side effects are usually mild. They are often more noticeable in the first four weeks of treatment and then begin to settle.

Possible side effects of TKIs

The most common side effects include feeling sick, diarrhoea, headaches, muscle and joint pains, dry skin, and fluid build-up, especially in the ankles or around the eyes. Side effects can usually be treated effectively. Tell your nurse or doctor if you have any side effects while taking imatinib.

Monoclonal antibodies (MAbs)

Monoclonal antibodies (MAbs) target specific proteins on the surface of the cells. They destroy the leukaemia cells by triggering the body’s immune system to attack the leukaemia cells and can cause the cells to kill themselves.

Rituximab is a monoclonal antibody that may be used to treat B-cell ALL. Alemtuzumab may be used to treat T-cell ALL.

MAbs can be given as a drip into a vein (intravenous infusion) or as an injection under the skin (subcutaneously). They may be given on their own or with chemotherapy.

Possible side effects of MAbs

Sometimes an MAb may cause an allergic reaction while it’s being given. This is more common with the first treatment, so you have it slowly over a few hours. The nurse will give you drugs to help prevent or reduce this. If you have a reaction they will treat it quickly.

Signs of a reaction can include:

  • flu-like symptoms (headaches, high temperature or chills)
  • feeling sick
  • pain in your back, tummy or chest
  • a rash
  • feeling itchy, breathless or unwell.

Tell your nurse straight away if you have any of these symptoms. Rarely, a reaction can happen a few hours after treatment.

Reactions are most common with the first dose, so, to reduce the risk of a reaction, the first infusion is given slowly over a number of hours. You'll be given medicines before the treatment that help to prevent or reduce any reaction.

Back to Targeted (biological) therapies explained

Angiogenesis inhibitors

Angiogenesis inhibitors prevent cancers from making new blood vessels. Without new blood vessels, the cancer cannot grow.

Cancer growth inhibitors

Cancer growth inhibitors block certain chemical signals within cells, which slows down or stops the growth of the cancer.

Monoclonal antibodies

Monoclonal antibodies can attach themselves to cancer cells to prevent them from growing.


Cancer vaccines help the immune system recognise cancer cells and destroy them.