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A new treatment approach in acute myeloid leukaemia

Acute myeloid leukaemia is the second most common form of childhood leukaemia, with 100 children diagnosed every year in the UK. Around a third of these young patients cannot be saved with existing treatments. Dr Mussai is taking forward a new approach to treatment that could offer new hope to children with this devastating disease.

We are grateful to the Aila Coull Foundation for providing the funding to support this project.


Project Details

  • Project Title

    A new treatment approach in acute myeloid leukaemia

  • Lead Researcher

    Dr Francis Mussai

  • Research Centre

    University of Birmingham

  • City & Institution Postcode

    Birmingham B15 2TT

  • Start Date


  • Duration


  • Grant Amount



Acute myeloid leukaemia (AML) is the second most common leukaemia of childhood, with 100 children diagnosed every year in the UK. Children aged less than two years at greatest risk of developing the disease.

Unlike the more common form of childhood leukaemia – acute lymphoblastic leukaemia (ALL) – from which most young patients can now be cured – a third of children with AML still cannot be saved.

Despite multinational research over the last 20 years, there have been no new drugs for the treatment of AML. A major challenge, therefore, is to find new drugs that can kill AML cells or that make AML cells more sensitive to the existing drugs used.

Dr Mussai and colleagues have previously shown that AML cells are reliant on a nutrient called arginine for critical cell processes that keep the AML cells alive. Arginine is an essential nutrient that is taken in as part of our diet.

An existing drug called BCT-100 can significantly lower arginine levels in the blood. In this pilot project, Dr Mussai and colleagues will investigate whether use of BCT-100 can either directly kill AML cells from children or make them more sensitive to standard chemotherapy drugs. The results could provide a completely new way to target AML and this work will lay the foundation for early phase clinical trials of the drug in children with AML.

What difference will this project make?

AML is the second most common cancer of the blood in children. Treatment is very intensive, often requiring inpatient admission for the majority of the treatment period. In high-risk disease a bone marrow transplant may be required. Despite all this, a third of young patients still lose their lives to the disease.

The ability of drugs to target essential nutrient use by cancer cells has already been shown to be successful in ALL, the more common form of childhood leukaemia. The use of a drug called Asparaginase reduces the amount of asparginine in the body and has led to significant improvements in the survival of children with ALL.

The results of this project could replicate this success in children with AML, providing a completely new way to target AML cells and laying the foundation for early phase clinical trials of a new therapy for children with this devastating disease.

About the Research Team

Dr Francis Mussai is a Consultant Paediatric Oncologist at Birmingham Children’s Hospital and a Senior Lecturer in Paediatric Oncology at the University of Birmingham.

The University of Birmingham has one of the largest blood cancer research departments in the UK and Birmingham Children’s Hospital houses the UK’s largest paediatric haematology department. This makes an ideal research environment to complete this project.

Dr Mussai is supported in this work by Professor Pamela Kearns, Consultant and Professor in Paediatric Oncology and Director of the CRUK Clinical Trials Unit. Professor Kearns brings extensive experience in the development of new drugs for clinical trials.

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