Dr Owen Williams and his team have identified an existing drug (mebendazole) that shows great promise in helping children with a life-threatening form of leukaemia. They will carry out further laboratory testing of the drug to prove its effectiveness, with the ultimate aim of rapidly initiating a trial of the drug with leukaemia patients.
Why this research is vital
Every year around 90 children in the UK are newly diagnosed with acute myeloid leukaemia (AML). Despite advances in treatment options, around a third of those children will lose their lives within five years of diagnosis. There is a desperate need to find new approaches that could save the lives of these children.
Blocking the development of acute myeloid leukaemia (AML)Recent progress in leukaemia research has identified a particular fault in the DNA of children with AML – a mistake in their body’s genetic instruction manual.
The genetic fault appears to fuel the aggressive development and progression of AML. The discovery of this cancer-advancing gene mutation is vital – if researchers can find a way to block its effects, they could stop a child’s cancer in its tracks.
In recent years, Dr Owen Williams and his team have screened existing medicines to see if they could be repurposed and used to effectively block the effects of the genetic anomaly in children with AML.
Remarkably, they found that mebendazole, a drug usually prescribed to treat parasitic worms, shows great promise in helping to stop the cancer progressing.
In this project, Dr Williams’ team will carry out further testing on mebendazole in the lab to see how effective it is at treating human AML cells, both by itself and in combination with other drugs.
By the end of the project, the team hope to be able to accelerate this treatment from the laboratory into clinical trial.
Impact of this project
Because the team is working with existing drugs that are already proven to be safe, they could be accelerated through to the clinic much faster than a new drug.
If this project is successful, it could give children with life-threatening AML and their families hope of a promising new treatment in the near future. This could give patients hope of a childhood free of cancer and allow their families to avoid the devastating news that nothing more can be done for their child.
This project is jointly funded by Sparks and charity partner Great Ormond Street Hospital Children’s Charity through their national research funding call.