Developing models to test new drug treatments for childhood leukaemia

11 May 2015
Dr Owen Williams, UCL Institute of Child Health

Despite improvements in survival, leukaemia remains one of the leading causes of death in childhood.

Dr Williams is focusing on the molecular biology of childhood leukaemia in order to identify new treatment approaches. He is working on the development of models that can accurately predict how certain childhood leukaemias respond to a new set of drugs. Ultimately this will improve the treatment options for children with leukaemia.

Amount of grant: £242,074 | Date of award: June 2014


Leukaemia is the most common childhood cancer. Advances in treatment mean that more than 80 per cent of young patients now survive but, despite this, leukaemia remains one of the leading causes of death in childhood.

Research by Dr Williams and colleagues at the Institute of Child Health, the research arm of Great Ormond Street Hospital, is based upon finding new treatments through understanding leukaemia at the molecular level. This has resulted in the discovery of faulty genes responsible for drug resistance in subclasses of acute leukaemias with poor outcomes.

In this new project Dr Williams and colleagues are focusing on a cell signalling pathway called STAT3. They have previously shown that the leukaemic cells in a common subtype of acute lymphoblastic leukaemia (ALL) have an exaggerated dependence on this pathway.

Together with other scientists, they have shown that many other childhood leukaemias are also dependent on this pathway. This means that the leukaemic cells can be specifically eliminated by drugs that block this normal pathway.

The team now aims to determine whether blocking the STAT3 pathway has therapeutic potential and to understand how it is controlled in leukaemia. This knowledge will be used to identify existing drugs, currently used for other diseases, which can interfere with the signalling pathway and potentially serve as new therapies.

They will focus their efforts on leukaemias that are difficult to treat using current chemotherapy, aiming to identify difficult to treat subtypes that are susceptible to STAT3 inhibition.

About the research team

Together we remain committed to finding novel cures for children who are in need of improved leukaemia therapies.
Dr Owen Williams
This project will involve the laboratories of Dr Owen Williams and Dr Jasper de Boer, in the Cancer Section of the UCL Institute of Child Health (ICH), and Dr Philip Ancliff, consultant paediatric haematologist at Great Ormond Street Hospital (GOSH).

The Williams and de Boer laboratories have an outstanding track record in childhood leukaemia research. Over the past decade they have developed models to investigate the molecular basis for leukaemia progression and susceptibilities to model therapies.

Together with Dr Ancliff, they have initiated a drug redeployment initiative at ICH and GOSH to identify and develop novel leukaemia therapies that can be ‘fast-tracked’ into the clinic.

Dr Ancliff will bring important clinical insight to this project and provide invaluable clinical experience to ensure the timely incorporation of new therapies into clinical trials and practice.

What difference will this project make?

The team will identify compounds that can eliminate leukaemia cells by inhibiting STAT3, while at the same time having minimal side-effects.

Existing drugs, already approved for clinical use, capable of STAT3 inhibition and consequent leukaemia eradication will be candidates for fast-track redeployment into leukaemia therapy.

Ultimately this will improve the treatment options for young patients with leukaemia, especially those with difficult to treat disease.

"This work is important as patients currently suffer from complications including drug resistance, therapy-related toxicity and relapse.  Novel therapeutic strategies are required to eradicate childhood leukaemia."
External reviewer

Read more: Treating childhood cancer | Side effects of treatment

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