Treatments for leukaemia

Learn about the common treatments for leukaemia and targeted therapies available to patients in the UK. Leukaemia is a type of blood cancer which affects white blood cells and starts in the bone marrow.

What treatments are available for leukaemia and other types of blood cancer?

Blood cancer is a type of cancer that affects the production and function of blood cells. The production of sufficient healthy and fully functional blood cells is essential to life and therefore treatment may be required when a person is diagnosed with blood cancer. Children and adults can be diagnosed with blood cancer however, the average age at diagnosis over 65 years of age.

A greater understanding of blood cancer and vastly improved treatments, over the last several decades, have resulted in significantly increased rates of survival for many of the different types of blood cancer, including leukaemia. Fortunately, many of these are now highly treatable.

The main types of treatment for leukaemia include watch and wait, chemotherapy, radiotherapy, stem cell transplant, immunotherapy, CAR-T therapy and targeted drugs. A more in-depth description of each type of blood cancer treatment is provided below.

The treatment or treatments recommended for blood cancer will depend on several factors. 

The factors include:

  • Type of blood cancer
  • Age of the person diagnosed
  • General health of the person diagnosed
  • How fast the cancer is progressing
  • The wishes of the patient

Recommended treatments may be one particular type of treatment or a combination of more than one. Immediate treatment may not be recommended for some people, particularly those with a slow-growing or chronic form of blood cancer.

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Common treatments for blood cancer are:

Some people diagnosed with blood cancer, particularly a slow-growing form, may not need treatment straight away, and some may never need treatment. The doctor may recommend ‘watch and wait’, usually considered when the symptoms are not troublesome, and the doctor believes there is no benefit to starting treatment straight away. Watch and wait is a safe option that avoids the side effects caused by many types of blood cancer treatment. Treatment may be recommended at a future date, if or when needed.

If watch and wait is recommended, regular appointments with the doctor will be necessary to check general health and symptoms, specifically to monitor whether the blood cancer remains stable or starts to progress.

Chemotherapy is the use of potent drugs or chemicals, often in combinations and given at regular time intervals, to kill or damage the blood cancer cells in the body. The chemotherapy drugs must be toxic enough to kill the blood cancer cells, it is therefore a harsh treatment option. All chemotherapy drugs interfere with cancer cells’ ability to grow or multiply. However, the drugs toxicity can harm the healthy cells as well which results in unpleasant side effects.

Side effects of chemotherapy1 tend to vary from person to person and can vary based on the amount or type of chemotherapy drugs. Some side effects can include:

  • Increased risk of infection
  • Breathlessness
  • Bruising easily
  • Diarrhoea
  • Fertility issues
  • Mouth sores/ulcers
  • Hair loss

Despite possible side effects, the ultimate goal of chemotherapy treatment is to damage or kill the blood cancer cells so there’s either no sign of illness (remission) or the disease’s progress is slowed. Chemotherapy can produce long-term remission or outright cure for many people, depending on the blood cancer type and the severity of the disease.

Chemotherapy is one of the most common treatments* for specific types of leukaemia such as acute lymphoblastic leukaemia (ALL). Chemotherapy treatment for leukaemia comes in different forms, which can include taking regular medication, injections or an intravenous drip into your bloodstream. 

Radiotherapy can be used to treat all types of blood cancer but more commonly used to treat Hodgkin lymphoma or Non-Hodgkin lymphoma.

Radiotherapy uses high-energy rays, such as x-rays, to destroy the blood cancer cells. Radiotherapy works by damaging the genetic material (DNA) within cells, which prevents them from growing and multiplying. Although the radiotherapy is directed at cancer cells, it can also damage nearby healthy cells. When radiotherapy is used for blood cancer treatment, it’s usually part of a treatment plan that includes drug therapy.

The treatment usually involves regular daily hospital visits to receive the radiotherapy but will not require a hospital stay. It may take several weeks to complete a course of radiotherapy.

Radiotherapy for leukaemia2 tends to be used in cases where leukaemia is causing bone pain, swollen lymph nodes or an enlarged spleen. The treatment may also be used if the stage of leukaemia is more advanced.

These are some of the side effects of radiotherapy2 which can include one or more of the following. It’s important to remember everybody reacts differently to treatment and your doctor will be able to offer further advice and guidance:

  • Fatigue
  • A skin reaction in the area treatment has been administered
  • Feeling nauseous
  • A drop in blood cell counts

Blood stem cells are produced in the bone marrow to become all the different types of blood cell the body requires. The blood stem cells are constantly dividing and maturing into different types of blood cells, replacing older and worn-out blood cells in the body.

Healthy stem cells are needed to live. When cancer or cancer treatments destroy the stem cells, stem cell transplantation (SCT) may be the best treatment option. Stem cell transplantation (SCT) is a procedure in which a patient receives healthy stem cells to replace faulty stem cells.

Before SCT, the patient receives high doses of chemotherapy, and sometimes radiation therapy, to prepare the body for transplantation. This is called “conditioning treatment.” After the stem cells are infused into the patient’s bloodstream, they travel to the bone marrow and begin the process of forming new, healthy blood cells including white blood cells, red blood cells and platelets. This process is called “engraftment.”

The main types of SCT are:

  • Autologous transplantation uses the patient’s own stem cells. These cells are removed, treated and returned to his or her own body after a conditioning regimen.
  • Allogeneic transplantation uses stem cells from a donor. A donor may be a family member or someone who is not related to the patient.

The main side effects of SCT3 or a bone marrow transplant are caused by chemotherapy, or if you are using targeted drugs. There are some side effects which are more common such as an increased risk of infection, increased risk of bleeding, nausea, diarrhoea, fatigue, loss of appetite or a sore mouth.   

If you are going through chemotherapy at the same time as receiving an SCT, you may experience additional side effects such as an increased risk of infections, nausea, fatigue or loss of appetite. It’s important to remember everybody reacts differently to treatment and your doctor will be able to offer advice.   

Immunotherapy, also known as biological therapy, uses the body’s own immune system to eradicate the blood cancer.

The immune system is designed to defend against disease-causing germs such as viruses and bacteria, including cancer cells, with the ultimate goal to keep us healthy. However, cancer can survive unchecked in the body because the immune system doesn’t recognise cancer as different. There are several different types of immunotherapies available to treat blood cancers, in recent years a very effective type has been developed called Chimeric Antigen Receptor T-Cell therapy (CAR-T therapy).

Side effects of immunotherapy4 can include:

  • Fatigue
  • Shortness of breath
  • Headaches
  • Feeling nauseous
  • Diarrhoea

It’s important to remember everybody reacts differently to treatment and your doctor will be able to offer advice.

CAR-T therapy is a new type of treatment that uses the immune system to kill cancer cells. In some cases CAR-T therapy has cured people where all other treatments have failed.

CAR-T therapy works by taking some T cells (blood cells that usually help to protect from infection and disease) out of the blood, genetically modifying them in a research laboratory so they are much better at recognising, finding and killing cancer cells, and then putting them back into the blood to eradicate the cancer.

The process of CAR-T therapy may take several weeks and has several stages:

  • T cells are taken from the blood, using a tube inserted into a vein in the arm. This may take two to three hours.
  • The extracted T cells are taken to a laboratory and genetically modified, turning them into CAR-T cells. This may take two to three weeks.
  • CAR-T cells are put back into the bloodstream via a drip. This process may last a few hours.
  • Once in the bloodstream, the CAR-T cells should now attack and eradicate the blood cancer cells.

Currently three types of CAR-T therapy are available in the UK. They are Kymriah (tisagenlecleucel), Yescarta (axicabtagene ciloleucel) and Tecartus (brexucabtagene autoleucel). In the UK CAR-T therapy has been approved to treat only a limited number of blood cancers and people, is not available for everyone diagnosed with blood cancer.

In England, Scotland Wales and Northern Ireland, CAR-T therapy has been approved to treat:

  • Children and adults up to the age of 25 with B-cell acute lymphoblastic leukaemia (ALL), whose first treatment has been unsuccessful.
  • Adults with types of Non-Hodgkin lymphoma called diffuse large B-cell lymphoma (DLBCL) or primary mediastinal large B-cell lymphoma (PMBCL), whose lymphoma has continued to grow after at least two treatments.

In England, Scotland Wales and Northern Ireland, CAR-T therapy can also be given through the Cancer Drugs Fund (CDF) and other patient access schemes to:

  • Adults with mantle cell lymphoma, whose lymphoma has continued to grow after two prior treatments including a Bruton’s tyrosine kinase (BTK) inhibitor such as ibrutinib.

CAR-T therapy side effects5 can include:

  • High temperature
  • Feeling nauseous or being sick
  • Difficulty breathing
  • Spells of dizziness
  • Headaches

With all forms of treatment, it’s always important to talk through options with your GP and/or cancer specialist.

Targeted drug therapy

Targeted therapies, sometimes called biological therapies, are cancer treatments that work by targeting the genetic changes that have occurred in cancer cells only, these genetic changes are not present in the normal cells. There are several types of targeted drug therapy which includes:

Monoclonal antibodies (MABs)

Antibodies are substances that occur naturally in the body and have a role in fighting infections.

Monoclonal antibodies are artificial antibodies that are made in the research laboratory. When given as treatment they work by recognising specific proteins found on the surface of the blood cancer cells. Much the same as antibodies produced by the healthy immune system to eradicate disease-causing viruses etc, the artificial antibodies seek out the cancer cells, and eradicate them.

Monoclonal antibodies may work in different ways. Some interfere with signals that a cancer cell needs to survive or divide. Others work by carrying a chemotherapy drug directly to a cancer cell. Some attach to cancer cells to make them more visible to the body’s immune system, which can then attack it.

Monoclonal antibody drugs all have names that end in “mab” e.g., Rituximab

Growth inhibitors (blockers)

There are many different type of cancer growth inhibitors that work in different ways. Our cells make chemicals called growth factors that function to control cell growth. Growth inhibitors work by blocking the growth factors that trigger the cancer cells to divide and grow.

Cancer growth blockers all have names ending in “ib”. Examples are: ibrutinib and imatinib (a type of tyrosine kinase inhibitor-TKI)


Surgery is rarely used to treat blood cancers, although a small number of people with lymphoma need to have their spleen removed.

Clinical Trials

Research studies involving testing new drugs and treatments using patients are called clinical trials. Clinical trials are important, because they are the only way to develop new treatments or improve existing treatments.

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