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Leukaemia is a type of blood cancer that usually affects white blood cells and bone marrow. White blood cells are an important part of your immune system that fight infection, and bone marrow is where blood cells like these are made.

There are many different types of leukaemia. Some types develop faster, and are known as acute leukaemia. These include acute lymphoblastic leukaemia (ALL), acute myeloid leukaemia (AML), acute premyelocytic leukaemia (APL), and B or T-cell acute lympoblastic leukaemia.

But each type of leukaemia acts differently, and will need to be treated differently.

Around 2,420 people are diagnosed with acute myeloid leukaemia (AML) every year. 

AML affects cells involved in the production of a family of blood cell called myeloid cells, which include red blood cells, platelets and certain types of white blood cells. 

We don’t have a lot of treatment options for AML, and it can be hard to treat, especially in older people who have a higher risk of developing the disease. Standard treatment is chemotherapy, and stem cell transplant is usually considered if chemotherapy fails or people relapse after initially responding to treatment. 

Because there are limited avenues of treatment, and at the moment we don’t have any targeted therapies for AML, this is a type of blood cancer where our research investment is high.

Our portfolio of research spans from finding where the roots of AML lie - so we can combat the disease right from the beginning - to testing new treatments for people living with AML. 


Clinical trials for people living AML

We are supporting a number of clinical trials for people living with AML. Research is taking place to test new drug combinations as quickly as possible to find potential new treatments for older people with AML.

Doctors are also looking at ways of making stem cell transplantation more effective for people, and to prevent AML from returning after this procedure.

Clinical trials for people living AML Select individually

FIGARO trial

A randomised trial of the FLAMSA-BU conditioning regimen in patients with acute myeloid leukaemia and myelodysplasia undergoing allogeneic stem cell transplantation 11/15/2013 10/16/2013 Non TAP Clinical trial Chief investigator Professor Charles Craddock, University Hospital Birmingham

This trial is looking at a reduced intensity conditioning regimen called FLAMSA-BU. Researchers think that this treatment could improve outcomes in older people because of reduced side effects and a lower risk of AML or MDS returning. FLAMSA-BU will be compared with standard intensity conditioning.

LI-1 Trial

LI-1 Trial: AML Pick A Winner Programme 01/10/2014 12/01/2011 Non TAP Clinical trial Chief investigator Professor Robert Hills, Cardiff University

People with AML and high risk MDS who are over the age of 60 are usually given cytarabine. In this trial, reasearchers want to know if cytarabine's effectiveness can be increased by giving the drug alongside newer chemotherapies or biological drugs.

ROMAZA trial

Phase I trial of romidepsin plus azacitidine in patients with newly diagnosed, relapsed or refractory acute myeloid leukaemia ineligible for conventional chemotherapy 01/01/2013 09/30/2013 TAP Clinical trial Chief investigator Professor Charles Craddock, University Hospital Birmingham

People with AML are usually given chemotherapy, but not everyone can have this type of treatment. Researchers think that combining azacitidine with romidepsin may help people with AML who can’t have standard chemotherapy treatment.

VIOLA trial

A phase I trial of combined azacitidine and lenalidomide salvage therapy in patients with acute myeloid leukaemia who relapse after allogeneic stem cell transplantation 09/01/2013 02/05/2014 TAP Clinical trial Chief investigator Professor Charles Craddock, University Hospital Birmingham

People with AML or MDS are usually treated with high doses of chemotherapy, followed by a stem cell transplant. But AML and MDS can come back after a transplant, and when it does it becomes much more difficult to treat. Researchers want to know if combining azacitidine and lenalidomide can help this group of people.

RAvVA trial

A randomised phase II trial of 5-azacitidine versus 5-azacitidine in combination with vorinostat in patients with relapsed acute myeloid leukaemia ineligible for salvage chemotherapy 08/01/2011 09/25/2012 TAP Clinical trial Chief investigator Professor Charles Craddock, University Hospital Birmingham

In this trial, researchers want to know if azacitidine given with vorinostat is better than azacitidine alone in people with AML or high risk MDS who are unable to tolerate intensive chemotherapy.

Finding out why people with AML relapse

Some people with AML can be cured with intensive chemotherapy, but others will unfortunately relapse. We still don’t fully understand why this is, and want to know the reasons for the variation in treatment response.

It is now clear that AML is particularly complex, with each leukaemia composed of different populations of cells that can vary widely in their genetic make-up. Research indicates that it is this remarkable variation of AML that influences how people with AML respond to treatment, and could be responsible for relapse.

One way to improve treatment for AML is to develop tests that can identify people with AML who are at risk of relapsing, so their treatment can be tailored accordingly.


Finding out why people with AML relapse Select individually

Pinpointing people who are at risk of relapsing

Understanding heterogeneity of treatment response in “standard risk” acute myeloid leukaemia 11/01/2016 Non TAP Grant Lead researcher Dr Richard Dillon, King’s College London

Using a test that picks up the mutation in the NPM1 gene, Dr Dillon wants to detect cells in the bone marrow that might be able to lie dormant and resist chemotherapy, causing relapse. This test was developed with Bloodwise funding, and is already helping doctors guide treatment for people with AML. This research will tell us more about the cells that harbour the NPM1 gene change.

Immunotherapy for AML

Immunotherapy is a type of treatment that uses the power of the body’s immune system to fight a disease.

There are numerous immunotherapies already being used to treat blood cancer, and many others that are being developed and tested in clinical trials.

These include:

  • Chimeric antigen receptor (CAR) and T cell receptor (TCR) therapy
  • Donor lymphocyte infusion (DLI)
  • Monoclonal antibody therapy
  • Reduced-intensity allogeneic stem cell transplantation

Part of our research is using a treatment approach called T cell receptor (TCR) therapy, which harnesses the body's immune system to fight cancer cells.

Cancer can only develop because faulty cells are able to hide from a person’s immune system. Scientists are using TCR therapy to re-educate a patient’s own immune cells to kill the cancer. To do this, a person’s immune cells - T-cells - are taken from their body and reprogrammed in a lab to recognise particular proteins on the surface of the cancer cells. The immune cells are then given back to the patient, so that the cells will kill the cancer cells.

A major advantage of this type of cell therapy over existing treatment options is that immune cells can recognise specific markers on cancer cells, providing a basis for the selective attack of cancer while avoiding damage to normal tissues.

We are also supporting a trial that is looking at a new monoclonal antibody that specifically targets a protein on AML cells. Early studies have shown the drug works well in a lab, so now researchers want to see if it can help people with AML who have not responded to the usual treatments, or have AML that has come back after treatment.

Preventing relapse after chemotherapy or stem cell transplants is really important because AML can be harder to treat in these circumstances. Our research is looking at using immunotherapy to boost the killing ability of the immune system so it can fight back against leukaemia, and we also want to know if using an immunotherapy can help to make stem cell transplants more effective.


Immunotherapy for AML Select individually

Fine tuning immune cells to be effective killers

Editing specificity and function to enhance T cell therapy of haematological malignancies 07/01/2013 Non TAP Grant Lead researcher Professors Hans Stauss and Emma Morris, University College London

In blood cancer, the patient’s own immune system is generally impaired so is unable to attack cancer cells. Professors Stauss and Morris are using TCR gene therapy to develop treatments to boost the immune response against blood cancers.


A first-in-human, first-in-class phase I dose escalation trial of humanized Anti-CD47 monoclonal antibody Hu5F9-G4 in acute myeloid leukaemia (AML) 03/27/2015 11/27/2015 Non TAP Clinical trial Chief investigator Professor Paresh Vyas, University of Oxford

Early studies have shown that a monoclonal antibody called Hu5F9-G4 can successfully eliminate AML cells in laboratory experiments. Researchers now want to see if Hu5F9-G4 can help treat people with AML in a clinical trial.

Breaking down the defences of leukaemia

In vivo analysis of the interactions between acute myeloid leukaemia, T cells and haematopoietic stem cells to inform the design of immunotherapy protocols 01/06/2016 Non TAP Grant Lead researcher Dr Cristina Lo Celso, Imperial College London

Using powerful microscopy, Dr Lo Celso and her team are analysing the evolving interactions between AML cells and T cells as disease develops. By understanding how AML cells switch off the immune system, researchers hope to target these mechanisms to help the immune cells to win against leukaemia.

Arming the immune system to fight against leukaemia

Pre-emptive immune therapy to prevent relapse of myeloid malignancies 08/01/2013 Non TAP Grant Lead researcher Professor Farzin Farzaneh, Kings College London

Professor Farzaneh and his team are developing two new treatments that change how the immune system responds to cancer and that will be given when AML is in remission to prevent relapse.

Optimisation of the graft versus leukaemia effect to improve the outcome of haematopoietic stem cell transplantation

Optimisation of the graft versus leukaemia effect to improve the outcome of haematopoietic stem cell transplantation 03/01/2013 Non TAP Grant Lead researcher Professor Paul Moss, University of Birmingham

Researchers led by Professor Moss want to investigate exactly how donor immune cells recognise and kill cancer cells. The aim of this research is to find new ways to increase the strength of GvL so that transplant treatments can be modified to the needs of individual people.


A phase II prospective trial of prophylactic donor lymphocyte infusions for the prevention of relapse post HSCT in patients with high risk myeloid malignancy 09/01/2016 Non TAP Clinical trial Chief investigator Dr Victoria Potter, Kings College Hospital, London

Stem cell transplantation may cure acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS), but unfortunately people can suffer a relapse after transplant. Researchers want to see if giving white blood cells from the stem cell transplant donor can be used after transplantation to prevent relapse.

Searching for new treatments for people with AML

Acute myeloid leukaemia (AML) is the most common aggressive blood cancer. It can be hard to treat, especially in patients over 65 years of age, and is often fatal within a year of diagnosis.

We still don’t have a lot of treatment options for AML. Standard therapy is chemotherapy, and stem cell transplant is usually considered if chemotherapy fails or people relapse.

Our research is looking for innovative ways to treat AML using targeted therapies, and to match existing therapies to the individual person so treatment has more success.

Searching for new treatments for people with AML Select individually

Discovering new leukaemia drugs

Targeting protein-protein interactions in leukaemia for novel therapeutics 03/01/2013 Non TAP Grant Lead researcher Professor Terence Rabbitts, University of Oxford

Professor Terence Rabbitts and his team are looking at three different leukaemia proteins called LMO2, RAS and MLL, which interact with each other to cause leukaemia. The team want to develop small antibody fragments that can block specific protein interactions, which could in the future lead to a new treatment option for people with AML. 

Personalising treatments for people with AML

The molecular genetics of acute myeloid leukaemia - stratified medicine to improve patient outcome 04/01/2014 Non TAP Grant Lead researcher Dr James Allan, Newcastle University

Dr James Allan wants to improve treatment options for people with AML. He will investigate using existing therapies more strategically based on the individual patient and leukaemia characteristics.

New treatment targets in AML

Targets for treatment in AML: Targeting the ROS axis 09/20/2015 Non TAP Grant Lead researcher Professor Richard Darley and Dr Alex Tonks, Cardiff University

Professor Richard Darley has identified an abnormality that is common to the majority of people with AML, which is the over-production of reactive oxygen species. The team have been looking at ways to exploit this situation, and are now refining these strategies for testing in a clinical trial.

Developing smarter and kinder treatments for AML

Functional dissection of transcriptional and epigenetic machinery in human leukaemia 09/01/2014 Non TAP Grant Lead researcher Professor Eric So, Kings College London

Abnormal gene expression is a common feature in leukaemia, and emerging evidence shows that proteins called histone modification enzymes play a key role in this process. Professor So aims to define the role of individual histone modification enzymes in the development and treatment of leukaemia, particularly AML. This could lead to the development of more effective but less toxic leukaemia therapies.

Targeting the unique features of leukaemia

Transcriptional control of normal and leukaemic blood stem/progenitor cells 03/01/2013 Non TAP Grant Lead researcher Professor Bertie Gottgens, University of Cambridge

Professor Bertie Gottgens and his team are using cutting-edge technologies to investigate networks that control the activity of genes in normal blood cells and AML cells, so that he can design new therapies that target leukaemia cells.

Understanding what drives AML

Our researchers want to understand what makes a healthy blood cell become a leukaemic cell, so that we can develop improved treatments. We know that damage to the genetic code carried by all blood cells is important. However, we don't fully understand what these changes do to healthy cells, and how changes in different genes work together to cause leukaemia.

And whilst most research focuses directly on understanding and treating blood cancer, there is a huge amount to learn from studying how healthy blood cells develop. If we can understand what should happen normally, scientists can really get a grip on what is going wrong in blood cancer.

One area we are particularly interested in is blood stem cells. These are a tiny population of self-maintaining cells that give rise to the billions of new blood cells that are produced daily. Many leukaemias are known to be sustained by transformed blood stem cells – sometimes known as ‘leukaemic stem cells’ or “cancer stem cells” - as a result of genetic changes they have acquired that permanently disrupt their normal behaviour. If we can learn about the mechanisms that turn healthy blood stem cells into leukaemic cells, we may be able to reverse this process. 

Our research is looking at blood stem cells in great detail, and how their development goes awry in leukaemia. We are also looking at where we can target these processes, which could lead to much needed treatments for AML.

Understanding what drives AML Select individually

Targeting the genes that cause AML

An investigation of the clonal and functional collaboration between IDH and SRSF2 mutations in acute myeloid leukaemia 08/01/2016 Non TAP Grant Lead researcher Dr Daniel Wiseman, University of Manchester

One in five AML patients has a mutation in genes called isocitrate dehydrogenase 1 (IDH1) or IDH2. People with IDH gene changes also have mutations in another gene called SRSF2. The team aim to understand how these mutations work together to cause AML. They will also profile and evaluate new candidate drugs which target the mutated form of IDH1 to see whether these successfully kill leukaemia cells, potentially leading on to clinical trials.

What turns healthy blood stem cells into leukaemic stem cells?

Mechanistic insights into aberrant transcriptional programming in acute myeloid leukaemia Non TAP Grant Lead researcher Professors Constanze Bonifer and Peter Cockerill, University of Birmingham

Professors Bonifer and Cockerill are investigating the complex processes that turn healthy cells into leukaemic cells. By understanding the causes of this transformation, they hope to find ways to prevent the development of AML - and to restore healthy blood cell production.

Improving care for people with inherited forms of MDS

The biology and management of familial myelodysplasia and leukaemia 06/01/2015 07/13/2017 Non TAP Grant Lead researcher Professor Inderjeet Dokal, Queen Mary University of London

By studying people with inherited MDS who are in the same family, Professor Dokal and his team hope to identify the fundamental steps that cause the disease. The team also want to determine the genetic variability of the disease, characterise new genes that drive MDS, and improve the management of people who have these inherited forms of MDS.

How do blood stem cells make decisions and how does this go wrong in leukaemia?

Understanding fate choice in normal and malignant haematopoietic stem cells 08/01/2015 Non TAP Grant Lead researcher Dr David Kent, University of Cambridge

Blood stem cells are responsible for making all blood cells, but in leukaemia the production of these cells goes wrong. Dr Kent and his team are working to find out how blood stem cells determine which blood cell they will develop into. This increased knowledge will be helpful in finding new treatments for leukaemia.

Similar research

Acute lymphoblastic leukaemia (ALL)

We are supporting a number of ALL research projects that are looking into what causes leukaemia, and how treatment resistance emerges and what we can do to reverse it.

Acute promyelocytic leukaemia (APL)

We are funding a project that wants to understand how APL develops, so we can have insight into why there is such a variation in response to treatment between people. This research could allow for treatments to be personalised based on an individual's APL profile.

Childhood acute lymphoblastic leukaemia (Ch-ALL)

Our research is looking into smarter and kinder treatments, and seeking ways to improve the diagnosis and outcome and treatments for children with leukaemia.

Childhood acute myeloid leukaemia (Ch-AML)

We have a childhood AML project that wants to know why some children with Down’s Syndrome are more prone to developing AML, and what we can do to stop this from happening. 

Chronic lymphocytic leukaemia (CLL)

Our portfolio of research is aiming to find out how CLL starts and what drives the disease, so we can find ways to prevent this. We also have a series of clinical trials that are looking at new ways to treat people living with CLL.

Chronic myeloid leukaemia (CML)

Our research is looking at new ways to eradicate CML completely, offering the chance of a real cure. And we want to find ways to improve the quality of life of people with CML who are taking these drugs.

Chronic myelomonocytic leukaemia (CMML)

We know we need kinder treatments for people with CMML, so we are supporting a trial that is looking at a drug called tefinostat, which could offer a less harsh treatment option. 

High-grade non-Hodgkin lymphoma (HGNHL)

We are supporting a portfolio of research, which aims to transform the way we diagnose and treat people with high-grade NHL, as well as searching for new treatments and tackling drug resistance.

Hodgkin lymphoma (HL)

We are supporting a programme of research, which could lead to a new targeted treatment for Hodgkin lymphoma. We are also running a trial that wants to reduce radiotherapy side effects in children, and another which is looking at a biologic treatment for people who cannot have standard therapy.

Low-grade non-Hodgkin lymphoma (LGNHL)

We are supporting research that hopes to improve the way people with mantle cell lymphoma are managed. We also have a project that hopes to find new ways to treat splenic marginal zone lymphoma.


Our researchers are making discoveries that will benefit people with both chronic and acute forms of leukaemia.


We want to know what starts myeloma, and find ways to prevent this from happening. We are working to improve current treatments and quality of life for people with myeloma, and to find a true cure for the disease. 

MPNs or MDS clinical trials

We have numerous clinical trials that are looking for new ways to treat people with MPN and MDS, so we can improve the lives of people living with these blood cancers.

Myeloproliferative neoplasms (MPN) and myelodysplastic syndromes (MDS)

We have a diverse portfolio of research that is advancing our understanding of MPN and MDS, that could lead to new treatments and improve the way people with these types of blood cancer are cared for.

Graft versus Host Disease (GvHD)

Our research is looking for new treatments for GvHD, and finding ways to optimise current treatments. And we want to find ways to boost the beneficial aspects of a stem cell transplant, whilst dampening down GvHD.