Cancer breakthroughs in children's medicine

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Today

Dual-use drugs Developing new drugs from scratch takes a huge amount of time, tenacity and money. So, in recent years, Dr Owen Williams and his team have taken a different approach. By examining a comprehensive catalogue of existing drugs, developed for anything from the common cold to HIV, they’re hunting for off-the-shelf treatments that could offer benefits for children with life-threatening cancers. Because the team is working with existing drugs already proven to be safe, they could be accelerated through to the clinic faster, cheaper and more easily than new drugs. This is known as drug repurposing. Central to its success is identifying key mechanisms causing the cancer and looking for existing drugs that could counteract them. Remarkably, several drugs – including mebendazole, usually prescribed to

treat parasitic worms – have already shown great promise in helping children with acute myeloid leukaemia (AML). These drugs effectively block key cancer-boosting processes inside cells. AML is diagnosed in around 90 children in the UK every year and, despite dramatic improvements in treatment, around a third will lose their lives within five years of diagnosis. While other researchers are pushing forward promising new treatments like CAR T-cell therapy, Dr Williams’ team hope existing drugs like mebendazole could offer ‘quick win’ approaches to help these children sooner. They’re also trawling the library for dual-use drugs to help children with other difficult-to-treat cancers, including the fatal brain tumour diffuse intrinsic pontine glioma (DIPG).

2020 Dr Owen Williams plans to initiate the UK’s first trial of an anti-worming drug to treat acute myeloid leukaemia (AML). 30

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