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Childhood leukaemia: investigating a new drug treatment

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Leukaemia is the most common cancer affecting around 500 babies, children and young people in the UK each year.[1,2] Thanks to advances in treatment, more than 80 per cent of children are now cured of their disease.[3] But unfortunately, certain subtypes of the disease are more difficult to treat – and the outlook for these children is not as positive. Dr Owen Williams at the UCL Great Ormond Street Institute of Child Health is exploring a new targeted drug combination, which he hopes will improve the outlook for young patients with a certain life-threatening type of acute leukaemia.

This project is jointly funded by Action Medical Research and LifeArc.

How are children’s lives affected now?

Leukaemia is a type of blood cancer that affects white blood cells. Changes to the DNA inside these blood cells cause them to grow too fast and take over the bone marrow. There are many different types – but leukaemia in children is almost always acute and develops faster than chronic forms, which usually affect adults.

“One subtype of acute leukaemia, which particularly affects babies under one, is caused by a particular DNA change in their cancer cells,” explains Dr Williams.

In these patients, part of the MLL1 gene has fused with another gene – which causes the affected blood cells to make a cancer-causing protein that is driving the disease.

“Even with the latest cutting-edge therapies, children with this type of leukaemia often do not respond to treatment – and sadly, many will lose their lives,” says Dr Williams. “We urgently need effective new approaches that can give hope to affected babies and their families.”

How could this research help?

“Ultimately, our goal is to develop a new treatment that will help to save the lives of these sick and vulnerable babies and children for whom no effective treatments exist,” says Dr Williams.

In previous laboratory experiments, the team has recently shown that a combination of two existing drugs can destroy the cancer-causing protein and stop the progression of the disease.

“This exciting breakthrough could be a game-changer in the treatment of the disease,” says Dr Williams

The researchers will now carry out further laboratory tests to explore the safety and effectiveness of this drug combination – and help to determine the best dose and treatment schedule.

“If our results are promising, they will help to inform the design of future trials in patients,” says Dr Williams.

As these drugs are already used to treat other unrelated conditions, this should help to accelerate the process of getting them into the clinic.


  1. Cancer Research UK Statistics: Children’s cancer incidence statistics: [website accessed 23 July 2019]
  2. Cancer Research UK Statistics: Young people’s cancer incidence statistics: [website accessed 23 July 2019]
  3. Cancer Research UK Statistics: Children’s cancer survival statistics: [website accessed 23 July 2019]

Research table

Project details

Project Leader Dr Owen Williams, PhD
Location Developmental Biology and Cancer Programme, UCL Great Ormond Street Institute of Child Health
Project Team Dr Jasper de Boer, PhD FHEA
Professor Ajay Vora, FRCPath
Professor Adele Fielding, ECFMG PhD MRCPath MRCP
Other Locations Haematology and Oncology Department, Great Ormond Street Hospital for Children
Research Department of Haematology, UCL Cancer Institute, University College London
Grant Awarded
Grant Amount £249,999
Start Date
End Date
Duration 36 Months
Grant Code (GN number) GN2820


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