https://www.gosh.nhs.uk/our-research/a-guide-to-research-and-innovation-at-gosh/cancer/

Cancer

Graphic of proposed new Children's Cancer Centre

We’ve come a long way in treating children’s cancers but it remains the leading cause of death in children aged five to 14. Four or more children die from cancer each week in the UK. We have big ambitions for cancer research at GOSH. Building a Children’s Cancer Centre that offers holistic, personalised and co-ordinated care as a national resource for children with rare and difficult-to-treat cancers will help advance our understanding of these diseases and developing new and better treatments.

Cancer research at GOSH

GOSH treats children with the rarest and most complex types of cancer. Some have exhausted their treatment options and are facing the most heart–breaking of outcomes. Many children with cancer at GOSH are on clinical trials as they desperately need new treatments. Researchers at the hospital are working on ground–breaking scientific ideas to make these treatments a reality. With some of the rarest cancers affecting only a few children a year, it’s vital our teams work together to gather data and understanding from as many patients as possible. There are incredible systems in place across the globe making it easier than ever for doctors and scientists to connect and share knowledge. Advances in genomic sequencing are not only helping us to understand a person’s genome, instead we can now also understand the genetic makeup of cancers. A UK-wide collaborative study called Stratified Medicine Paediatrics – led from the Institute of Cancer Research and UCL GOS ICH – has been sequencing all childhood relapse cancers and, via a National board led by Professor Darren Hargrave at GOSH, feeds back this genetic information to clinicians all over the UK. This allows a targeted therapies approach, with several led by Professor Hargrave showing early promise in paediatric brain tumours.

Dr Darren Hargrave

Professor Darren Hargrave

“A decade ago, our primary diagnostic tool for cancer was looking at the cells under a microscope. Genetics is revolutionising the way we diagnose and treat these and other diseases. Studies have shown that for virtually every ‘type’ of cancer, there are many sub types, each with different genetic characteristics. That’s telling us incredibly valuable things about which existing treatments are likely to work for which types, as well as giving us new ideas for treatments.”

Image of a young child wearing a white dress and headscarf, with a blue headband of flowers, running. Behind her is young boy running after her.

Ayaan's story

Ayaan (photographed above) was diagnosed with acute myeloid leukaemia (AML) in 2020 after becoming unwell with a fever, unexplained bouts of exhaustion, and a lack of appetite. Her family were told she had a 60 per cent chance of survival but, after advanced genomic testing, the Genomics Laboratory Hub based at GOSH found a gene that was associated with a different cancer, acute promyelocytic leukaemia (APL). This changed things drastically for the family. The new diagnosis called for less intensive treatment, so she could be at home with her family, and APL has a long-term survival rate of more than 90 per cent.

Drug repurposing

Developing completely new drugs is an expensive and time–consuming process, so researchers at GOSH are also working to understand if there are drugs that are already approved for other conditions that could be used to treat cancer. This is known as drug repurposing. The team, led by Professor Owen Williams, are just one group working on a different approach. They have already shown that mebendazole, a drug originally prescribed to treat parasitic worms, shows promise in treating children with acute lymphoblastic leukemia.

CAR-T cell therapy research