Treatment

Updated March 20, 2025

There are a variety of different treatments that you or your child may receive for acute lymphoblastic leukemia. It is likely treatment will begin rapidly after receiving a diagnosis.

Chemotherapy

Chemotherapy is the most common form of treatment for acute lymphoblastic leukemia. Chemotherapy works by killing rapidly dividing cells such as cancer cells. Treatment may involve the use of one or more chemotherapy drug at once, and often these agents are given straight into the bloodstream. The type of treatment you receive will depend on a number of factors such as your age and fitness levels.¹

Phases of treatment

1. Induction phase with high doses of therapy

May last 1 or 2 months. The aim of the induction phase is to reduce the number of leukemia cells to a point when they are no longer detectable in bone marrow samples (called complete remission). After this point, the normal blood cells in the marrow can regrow. While achieving a complete remission is a positive sign, it does not necessarily mean the disease is cured, as leukemia cells may be present at very low levels or in other parts of the body.²

2. Consolidation with high doses of therapy

May last 1 or 2 months. The consolidation phase is designed to stop the acute lymphoblastic leukemia coming back and to destroy any leukemia cells that may be present in the blood but that are in such small numbers they do not show up on tests.¹

3. Maintenance

Maintenance is a less intense treatment phase that can last up to 2 years. It aims to maintain remission by killing the few leukemic cells that have evaded previous rounds of therapy. Maintenance therapy uses lower doses of chemotherapy, so the side effects should be less during this phase, allowing a return to normal activities.¹

Common chemotherapy agents used include:

• Asparaginase
• Vincristine
• Cyclophosphamide
• Cytarabine
• Doxorubicin
• Methotrexate
• Steroids such as dexamethasone, prednisolone, and hydrocortisone taken before or alongside chemotherapy¹

Targeted treatment

These types of treatment target specific parts of cancer cells and typically target cancer cells only if certain genetic changes are present.

Examples of genetic changes for which targeted treatment is available:

The Philadelphia chromosome is the name given to the product of swapping specific genetic material between chromosomes 9 and 22. This results in the creation of a leukemia-specific gene called BCR::ABL1. Leukemia cells with this change are sensitive to treatment by tyrosine kinase inhibitors such as³:

• Imatinib
• Dasatinib
• Ponatinib
• Nilotinib
• Bosutinib

Activation of various kinases can occur in some subtypes of acute lymphoblastic leukemia, including in what is referred to as ‘Philadelphia-like’ acute lymphoblastic leukemia, and heretyrosine kinase inhibitors can also be included in the treatment. Ruxolitinib is such an inhibitor that is currently being tested in clinical trials.

In leukemia with changes within the KMT2A gene, a protein called menin interacts with KMT2A proteins and promotes the growth of leukemia cells. Menin inhibitors are drugs that block the interaction between menin and KMT2A, preventing the growth of leukemia cells.

Revumenibis a menin inhibitor that is approved in the US for the treatment of patients who have acute leukemia and changes within their KMT2A gene, where the leukemia has returned or is no longer responding to treatments. Currently, menin inhibitors and revumenib are only used to treat patients whose leukemia has returned after multiple rounds of chemotherapy.

Immunotherapy

Immunotherapy can be considered to be a specialized form of targeted therapy that uses specifically designed antibodies to treat acute lymphoblastic leukemia. Immunotherapy aims to encourage the patient’s immune system to target and kill leukemia cells.⁴

The two main immunotherapeutic agents you may be treated with are³:

• Blinatumomab
• Inotuzumab ozogamicin³

Blinatumomab

Blinatumomab is a special antibody used to treat some subtypes of B-cell acute lymphoblastic leukemia. Blinatumomab binds to a molecule on the surface of leukemic cells called CD19, as well as a molecular on the patient’s own normal Tcells. This enables the patient’s own immune system to kill the leukemic cells. Treatment usually occurs in a hospital or clinic because it is a relatively new therapy and it is important to document the side effects. Treatment of patients with B-cell acute lymphoblastic leukemia with blinatumomab is rapidly becoming the gold standard treatment and is very likely to revolutionise the treatment of this disease.

Inotuzumab ozogamicin

Inotuzumab ozogamicin is another unique antibody which is used to treat some subtypes of B-cell acute lymphoblastic leukemia after unsuccessful prior chemotherapy. It works by delivering the chemotherapy drug to the leukemia cells and killing them. 

Chimeric antigen receptor (CAR) T cells

CAR T-cells are a recent advancement in the treatment of acute lymphoblastic leukemia. White blood cells from the patient are taken and engineered to specifically kill leukemic cells. These white blood cells are now called CAR T-cells and are multiplied before being given back to the patient. Tisagenlecleucel is a CAR T-cell product that can be used to treat acute lymphoblastic leukemia,⁴ it has been approved for use in children and young adults up to age 25 to treat B-cell acute lymphoblastic leukemia that has returned or is no longer responding to treatment.

Brexucabtagene autoleucel is approved to treat adults with B-cell acute lymphoblastic leukemia that has returned or is no longer responding to treatments.

Obecabtageneautoleucel is a CAR T-cell therapy that is approved in the US to treat adults with B-cell acute lymphoblastic leukemia that has returned or is no longer responding to treatments.

Radiation therapy or radiotherapy

High doses of targeted radiation, normally called X-rays, can be used to treat cancer, and this technique is called radiotherapy. This may be performed on the brain or spinal cord if the leukemia appears to have spread to these areas. Alternatively, radiotherapy may be used over the whole body (known as total body irradiation) prior to a stem cell transplant.

Stem cell or bone marrow transplant

This procedure is used following chemotherapy or radiotherapy. High intensity treatment is used to kill the leukemic cells in the bone marrow (the spongy part of the bone); however, normal blood cells are killed too.

A transplant is used to replace the blood cells with healthy cells that function correctly. The healthy cells are normally provided by a donor. In a transplant, these healthy cells can be taken from the bone marrow or from the bloodstream.

Side effects³

Although many treatments for acute lymphoblastic leukemia are very good at killing cancer cells, some normal healthy cells may be damaged or killed during treatment as well. This may result in side effects.

For chemotherapy, traditional side effects associated with treatment are:

• Nausea and vomiting
• Loss of appetite
• Fatigue
• Mouth ulcers
• Diarrhea
• Hair loss
• Infertility
• Skin rashes

Immunotherapy may increase the risk of infection along with:

• Headaches
• Fever
• Muscle aches
• Dizziness
• Bleeding
• Feeling and being sick⁴

CAR T-cell therapy may result in serious side effects, for this reason it is only given in specialized medical centers by trained staff. The most common side effects include:

• Cytokine release syndrome (CRS): symptoms including fever, chills, difficulty breathing, severe nausea, vomiting, diarrhea, severe muscle or joint pain, and feeling dizzy
• Nervous system problems: can present as headaches, changes in consciousness, confusion or agitation, seizures, trouble speaking and understanding, or loss of balance
• Infections
• Low blood cell counts