Research into acute myeloid leukaemia (AML)

Researchers around the world are looking at better ways to treat acute myeloid leukaemia (AML) and manage side effects. Go to Cancer Research UK's clinical trial database if you are looking for a trial for AML cancer in the UK.

Talk to your specialist if there are any trials that you think you might be able to take part in. 

Some of the trials on this page have now stopped recruiting people. It takes time before the results are available. This is because the trial team follow the patients for a period of time and collect and analyse the results. We have included this ongoing research to give examples of the type of research being carried out in AML. 


Chemotherapy uses anti cancer (cytotoxic) drugs to destroy cancer cells. The drugs circulate throughout the body in the bloodstream. It is the main treatment for AML. There are many trials looking at improving chemotherapy treatment for people with AML. 

Trials are looking into:

  • different combinations of chemotherapy 
  • whether chemotherapy can prevent leukaemia from coming back 
  • reducing chemotherapy side effects
  • how chemotherapy affects quality of life 


A transplant allows you to have high doses of chemotherapy and other treatments. Your team either collect:

  • your own stem cells
  • or a donor's stem cells

After the treatment you have the stem cells into your bloodstream through a drip. The cells find their way back to your bone marrow where they start making blood cells and your bone marrow slowly recovers. 

Mini transplants 

The mini transplant (or reduced intensity conditioning transplant) gives lower doses of chemotherapy than usual transplants. It makes use of the graft versus leukaemia effect. The chemotherapy doses you have are too low to destroy your own bone marrow. You have just enough chemotherapy to damp down your bone marrow until the transplanted donor cells have settled into it and started to produce blood cells.

The aim is that you develop a mild graft versus host disease (GvHD) reaction so the donor cells can attack and kill the leukaemic cells.

Trials for AML are looking into:

  • how well mini transplants work
  • different chemotherapy drug combinations before transplants

Half matched (haploidentical) transplants

Doctors can't find a match for about 1 in 3 people who need a transplant. The UK Haplo study is looking at using a half matched transplant in this situation. In a half matched transplant, the donor is at least a 50% match with the person having the stem cell transplant.

Researchers want to find out:

  • how well high dose chemotherapy works with a half matched stem cell transplant
  • how safe it is
  • about side effects
  • how it affects quality of life

Cord blood stem cell transplants

Trials are using blood stem cells collected from the umbilical cords of newborn babies. They are looking into whether they can these stem cells with different doses of chemotherapy and what the side effects are.

AML treatment in older people

Treatment for AML varies with age. You have to be very fit to get through some of the intensive treatments, so doctors don't generally use them for older people. The older you are, the less likely you are to be fit enough. The good news is that as we get better at managing side effects, intensive treatments are being used more for older people. 

AML trials are looking into:

  • finding better treatment combinations for older people 
  • whether intensive or non intensive treatment works better
  • different chemotherapy drugs
  • whether targeted therapies can help alongside chemotherapy
  • whether new treatments are safe for older people

Targeted cancer drugs

Targeted cancer drugs work by ‘targeting’ those differences that help a cancer cell to survive and grow. There are many different types of targeted drugs with different ways of working. Some seek out and destroy cancer cells. Others help the immune system to attack the cancer. Researchers are looking into:

  • whether targeted drugs alongside chemotherapy will improve treatment
  • whether certain drugs work best for young people, children or older people
  • different treatment options for people who can't have intensive treatment


Immunotherapies can boost the body's own immune system to fight off or kill cancer cells. Researchers in one trial are creating and testing a personal vaccine. This vaccine is made by altering your own leukaemia cells and injecting them back to trigger their immune system. 

CAR T-cell therapy is another treatment that uses your own immune system to treat leukaemia. Doctors remove a type of white blood cell, called T cells. These cells are very good at helping fight infection, but they aren't so good at telling the difference between a normal cell and a cancer cell. Doctors then change the T cells in the laboratory and give them back to you through an infusion. The aim is that the altered T cell recognise and attack any leukaemia cells.

This treatment is now used for children with a type of acute lymphoblastic leukaemia. It is in trial for children with other blood cancers, including acute myeloid leukaemia.

Last reviewed: 
18 May 2020
Next review due: 
18 May 2023
  • Cancer Research UK Trials database
    Accessed September 2019

  • Acute Myeloblastic Leukaemia in Adult Patients: ESMO Clinical Practice Guidelines
    M Fey and C Buske, 2013
    Annuals of oncology. Volume 24, Issue 6