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Acute myeloid leukaemia research

Men and women discussing acute myeloid leukaemia

Find out about research into the causes, prevention and treatment of acute myeloid leukaemia. You can find the following information


A quick guide to what's on this page

Acute myeloid leukaemia research

All treatments must be fully researched before they can be adopted as standard treatment for everyone. This is so that we can be sure they work better than the treatments we already use. And so we know that they are safe.

First of all, treatments are developed and tested in laboratories. Only after we know that they are likely to be safe to test are they tested in people, in clinical trials.

Research is looking into new chemotherapy drugs, stem cell transplants, biological therapies and treating AML in older people. Cancer Research UK supports a lot of UK laboratory research into cancer and also supports many clinical trials.


CR PDF Icon You can view and print the quick guides for all the pages in the treating AML section.



Why we need research

We must fully research all potential treatments before we can adopt them as standard treatment for everyone. This is so that

  • We can be sure they work
  • We can be sure they work better than treatments that are available at the moment
  • They are known to be safe

First of all, treatments are developed and tested in laboratories. For ethical and safety reasons, experimental treatments must be tested in the laboratory before they can be tried in people. If a treatment described here is said to be at the laboratory stage of research, it is not ready for patients and is not available either in the NHS or in private healthcare organisations.

Cancer Research UK supports a lot of UK laboratory research into cancer and also supports many clinical trials. Clinical trials test treatment in patients and there are 4 phases of trials.

If you are interested in taking part in a clinical trial for acute leukaemia, visit our searchable database of clinical trials in the UK. If you are interested in a particular trial that is recruiting, you can print it off and take it to your own specialist. If the trial is suitable for you, your doctor needs to refer you to the research team. The database also has information about closed trials and trial results. Most major leukaemia treatment centres are continually involved in clinical trials.

Here is a video on what it's like to take part in a clinical trial:

View a transcript of the video (Opens in a new window)


Chemotherapy drugs

Trials are looking at different combinations of chemotherapy. They're looking at

  • whether chemotherapy can prevent AML from coming back (recurrence)
  • learning about chemotherapy side effects
  • new chemotherapy drugs
  • different chemotherapy drug combinations
  • how quality of life is affected

Find out more about chemotherapy trials for AML


Mini transplants

Bone marrow and stem cell transplants can work well for acute leukaemia. But the intensive chemotherapy can be so difficult that only very fit patients can cope with it. Now doctors are looking at a new, less harsh type of transplant.

Doctors know that leukaemia is less likely to come back in people who have a donor transplant and get a reaction called graft versus host disease (GVHD). In GVHD the donor blood cells attack the patient's own cells, including the leukaemia cells. Doctors call this the graft versus leukaemia (GVL) effect.

The mini transplant (or reduced intensity conditioning transplant) 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 marrow until the donor cells have settled into it and started to produce blood cells. 

The aim is that you develop mild GVHD so the donor cells can attack and kill the leukaemic cells. Although this treatment helps more people to live longer than a full transplant, you still need to be fairly fit. The GVHD can be tough to cope with.

Different trials for AML are looking into

  • how well less intense transplants work
  • different chemotherapy drug combinations before transplants

Find out more about mini transplants for AML


Half matched stem cell transplants

Doctors can't find a match for about a third of those needing a transplant. The UK Haplo study is looking at an option for these people. This is a half matched transplant, where the donor is at least 50% match with the person having the stem cell transplant. 

The aim is to find out

  • how well a new drug works with a half matched stem cell transplant
  • how safe it is
  • about side effects 
  • if quality of life is affected

Find out more about the UK Haplo study


Cord blood stem cell transplants

Bone marrow and stem cell transplants for AML use high dose chemotherapy, and sometimes total body radiotherapy. Bone marrow or stem cell transplants give the best chance of controlling the AML for a long time in some people. But the high dose treatment can also cause severe side effects. Doctors are trying to find ways of improving these treatments. 

Trials are using the 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.

Find out more about cord blood stem cell transplants


AML 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 the effects, intensive treatments are being used more for older people.

Doctors think about treatment and the possible effects individually for each patient. You can be in your 50s and not be as fit as some people who are in their 60s or even older. Whether you can have a transplant also depends on whether you have a brother or sister fit enough to be a donor. As you get older, the chance of having a donor who is fit enough gets lower.

AML trials are looking into

  • different treatment combinations to improve treatment for older people
  • whether intensive treatment or non intensive treatment works better
  • different chemotherapy drugs
  • whether biological therapies can help with chemotherapy
  • whether new treatments are safe for older people

For more information, read about the different trials on treatment for older people with AML


Diagnosing fungal infection

Aspergillosis is a chest infection caused by the aspergillus fungus. Chemotherapy and stem cell transplants weaken your immune system, which means you have a higher risk of getting aspergillosis. 

At the moment, the only way to be sure you have this infection is to have a test called a bronchoscopy. This involves putting a tube down your windpipe and into your lungs to take samples. You have a local anaesthetic or a drug to make you drowsy before the bronchoscopy. But it can still be uncomfortable.

A trial is testing 2 new ways of checking for aspergillus. One is a blood test and the other is a breath test. Researchers want to see how good these tests are at finding aspergillus infection in people with acute leukaemia.


Biological therapies

Biological therapies are treatments that can control the growth of cancer cells by changing or blocking chemical pathways in the cells. Biological therapies studied in acute leukaemias include

There are different trials happening within these types of biological therapies. They are looking into

  • using biological therapies alongside chemotherapy
  • comparing biological therapies with chemotherapy to see what works best
  • if certain drugs work best for young people, children and older people
  • what the side effects are 
  • different treatment options for people who can't have intensive treatment
  • ways to stop leukaemia from coming back
  • a new vaccine to boost the immune system and kill of leukaemia cells

Find out more information on biological therapy trials for AML


For more information

Find out about

Experiences of taking part in a trial

Clinical trials database

Understanding clinical trials

Phases of clinical trials

Chemotherapy for AML


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Updated: 29 June 2016