Targeted therapies 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 are used to treat Philadelphia positive acute lymphoblastic leukaemia (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.

Tell your nurse or doctor if you have any side effects. They can usually be treated effectively.

Monoclonal antibodies (MAbs)

Monoclonal antibodies 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.

MAbs may be given on their own or with chemotherapy and can be given as:

  • a drip into a vein (intravenous infusion)
  • an injection under the skin (subcutaneously).

MAbs can sometimes cause an allergic reaction. This is most common with the first dose. To reduce the risk of a reaction, the first infusion is given slowly. You‘ll be given medicines before the treatment that help to prevent or reduce any reaction. Tell your nurse or doctor if you feel unwell while having MAbs.

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

Inotuzumab and blinotumumab are newer monoclonal antibodies being used to treat ALL. They are given to people whose leukaemia has not responded to treatment or whose leukaemia has come back after treatment. They are usually given as part of a clinical trial. You can talk to your specialist doctor or nurse about whether these treatments might be right for you.

Back to Targeted (biological) therapies explained

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.