Developing new safer treatments for children with acute myeloid leukaemia

Acute myeloid leukaemia (AML) is an aggressive disease that is responsible for roughly 40% of deaths from blood cancer in children every year

Project Details

  • Project Title

    MYB-directed therapy for paediatric AML

  • Lead Researcher

    Professor Owen Williams

  • Research Centre

    UCL Cancer Institute

  • City & Institution Postcode

    London, WC1E 6JD

  • Start Date

    1 April 2024

  • Duration

    36 months

  • Grant Amount


Owen Williams researcher in white lab coat


Acute myeloid leukaemia (AML) is an aggressive disease that is responsible for roughly 40% of deaths from blood cancer in children every year. Although modern medicine has made dramatic advances in the treatment of this disease, further improvements cannot be achieved by increasing the intensity of current therapies due to the unacceptable toxicities this would entail. For this reason, research has focused on trying to block the action of different cancer genes that are responsible for this disease. However, it is known that many different cancer genes can cause AML and so a therapy that blocks the action of any one of these will only be useful in a proportion of cases. Another approach is to find cellular pathways that may be hijacked in common by the different cancer genes. Therapies designed to block these could then be used across a broad range of different AML patients. Professor Owen Williams, his team and others have shown that one such pathway does exist. It is controlled by a molecule called MYB and laboratory research has shown that blocking this pathway results in the death of blood cancer cells from different types of AML. The next step is to find safe and effective drugs that can block this pathway in children suffering from AML.

Professor Williams and his team plan to establish a new treatment for children suffering from AML, based on their recent research. This project aims to produce data that will be sufficient to initiate a phase I clinical trial for this devastating disease. The team plan to investigate in more detail the cellular mechanisms hijacked in this type of cancer and find ways in which these can be blocked. The overall goal is to develop a new and safe treatment for AML in children.

The team have recently published research showing that the drug mebendazole, which is used in millions of children worldwide to treat parasitic worm infections, kills AML cells by blocking the action of MYB. They are currently working out how mebendazole can be used in the clinic by testing which of the current AML treatments it can be used together with. This project is based on further research that undertaken looking at ways in which AML cells can resist mebendazole. This work found two pathways that can be safely controlled to increase the killing of AML cells by mebendazole. Professor Williams and his team propose testing these modified treatments in a broad range of blood cancer cells from different types of AML. They also plan to continue their work at finding new ways in which to improve the effectiveness of mebendazole in treating AML in children. Since there are many laboratories and companies that are developing new treatments that block the action of MYB, the team also plan to use a laboratory model to find cellular pathways in AML cells that affect their ability to grow and survive when MYB is blocked.

What difference will this project make?

This research is crucial to the rapid translation of mebendazole into the clinic for children suffering from AML but also to any new therapy that emerges in the near future designed to block MYB in AML. This project aims to produce sufficient evidence to support the start of a Phase I clinical trial for mebendazole, in combination with other treatments, in children suffering from AML. If this is successful, it will make an impact on the treatment of these children, potentially improving their treatment outcomes without increasing the toxicities they are exposed to.

About the Research Team

Professor Owen Williams, has run a research group in the UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) for over 20 years, investigating how blood cancer forms in children and how this knowledge can be used to design new treatments for this disease. His group works in close collaboration with clinical colleagues in both Great Ormond Street Hospital (GOSH) and University College London Hospital (UCLH) to identify new suitable treatments that can be safely used in children with AML.

The co-applicants on this project, Dr David O’Connor (GOSH, UCL Cancer Institute), Dr Jack Bartram (GOSH) and Dr Rob Sellar (UCLH, UCL Cancer Institute) are consultant haematologists and clinical scientists with first hand expertise in treating children with AML and in laboratory research techniques that are vital for this project.

The team will also collaborate with Professor Marc Mansour (UCL GOS ICH, UCL Cancer Institute, UCLH) who is a group leader and consultant haematologist, who’s laboratory and clinical expertise will also be vital for this project. This team already works together and has made important contributions to the study, diagnosis and development of treatments for children and young adults suffering from blood cancer.

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