Clinical trials and acute leukaemia

Most people in the UK have treatment for acute myeloid leukaemia (AML) or acute lymphoblastic leukaemia (ALL) as part of research called clinical trials.

What are clinical trials?

Most people in the UK have treatment for acute myeloid leukaemia (AML) or acute lymphoblastic leukaemia (ALL) as part of research called clinical trials. These are usually large national and international studies that aim to find out more about leukaemia and treatment. They use drugs and treatments for leukaemia that are known to work well. But they may give them:

  • in different combinations
  • with newer types of drugs.

Clinical trials may:

  • test new treatments, such as new chemotherapy drugs
  • look at new combinations of existing treatments, or change the way they are given to make them more effective or reduce side effects
  • compare the effectiveness of drugs used to control symptoms
  • find out how cancer treatments work
  • find out which treatments are the most cost-effective.

Trials are the only reliable way to find out if a different type of chemotherapy, radiotherapy, or other treatment is better than what is already available.

Taking part in a trial

Usually, several hospitals around the country take part in these trials. It’s important to remember that some treatments look promising at first but later are shown to be less effective or have side effects that outweigh the benefits.

Your doctor or nurse will explain this and what is involved in a trial before you make any treatment decisions.

If you decide not to take part in a trial or change your mind at any time, your doctor will respect your decision and you do not have to give a reason. However, it can help to let them know your concerns so that they can give you the best advice. There will be no change in the way that you are treated by the hospital staff, and you will be offered the standard treatment for your situation.

We have more information about clinical trials.

Blood and bone marrow samples

Blood and bone marrow samples are taken to help make the right diagnosis. You may be asked for your permission to use some of your samples for research into cancer. If you take part in a trial you may also give other samples. These may be frozen and stored for future use when new research techniques become available. Your name will be removed from the samples so you cannot be identified.

The research may be done at the hospital where you are treated, or at another one. This type of research takes a long time, and results may not be available for many years. The samples will be used to increase knowledge about the causes of leukaemia and its treatment. This will hopefully improve the outlook for future patients.

About our information

  • AML References

    Below is a sample of the sources used in our acute myeloblastic leukaemia (AML) information. If you would like more information about the sources we use, please contact us at

    British Committee for Standards in Haematology. Milligan DW et al. Guidelines on the management of acute myeloid leukaemia in adults. British Journal of Haematology. 2006. 135: 450–474. 

    Fey MF and Buske C. Acute myeloblastic leukaemia in adult patients: ESMO clinical practice guidelines. Annals of Oncology. 2013. 24 (Supplement 6): vi138-vi143.

    National Institute for Health and Care Excellence. Blood and bone marrow cancer. (accessed July 2018).

  • Reviewers

    This information has been written, revised and edited by Macmillan Cancer Support’s Cancer Information Development team. It has been reviewed by expert medical and health professionals and people living with cancer. It has been approved by Senior Medical Editor, Dr Anne Parker, Consultant Haematologist.

    Our cancer information has been awarded the PIF TICK. Created by the Patient Information Forum, this quality mark shows we meet PIF’s 10 criteria for trustworthy health information.