Fast-tracking a new leukaemia treatment towards clinical trial

Dr Owen Williams, Professor of Cancer Biology at the UCL Great Ormond Street Institute of Child Health, and his team have identified an existing drug that shows great promise in helping children with acute myeloid leukaemia.

Every year, around 90 children in the UK are 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.

One of the latest research projects funded by Great Ormond Street Hospital Children’s Charity aims to find more treatments for children with AML, by ‘repurposing’ existing drugs.

Dr Owen Williams and his team have identified an existing drug (mebendazole) that shows great promise in helping children with AML. This year, 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.

A vital discovery

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.

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.

Next steps 

Further testing on mebendazole will be conducted 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.

Because the team are working with existing drugs 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.

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