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Case study

Using mice to mimic childhood cancers

Children with cancer have a good survival rate overall, but treatments often have long-term side-effects, and a significant proportion of patients develop aggressive disease which is still often fatal. The Institute of Cancer Research is committed to understanding the biology of children’s cancers so we can design kinder, more effective treatments – and we do this work by studying children’s cancer in mice.

Professor Louis Chesler, a paediatric cancer researcher at the ICR and an honorary consultant at The Royal Marsden, is uncovering the genetic weaknesses of children’s cancers, and discovering and developing new targeted drugs for cancers that respond poorly to existing treatments.

One example is neuroblastoma, a nerve tumour which is a major cause of death in young children. Most children with the disease have a high-risk, aggressive form at the time of diagnosis that is very difficult to cure and over half of these children relapse following treatment.

Professor Chesler works with mice to gain a better understanding of the complex biology of what drives this childhood cancer. The mice are genetically engineered to have the same mutations found in patients with this disease, and faithfully mimic this high risk group of neuroblastoma patients. Professor Chesler has used mouse modelling to discover that a combination of a mutation in a gene called ALK and an increase in the number of copies of a gene called MYCN drives a particularly aggressive form of the disease, which is crucial in our ultimate goal of finding potential targeted therapies.

Our mouse work is an essential part of our research to discover new treatments for cancer. With these models we can conduct trials of potential new cancer drugs in mice – using a unit we have set up called a mouse hospital. We can find which drugs have the best results in the mice, shrinking the tumours and ultimately killing the cancer cells. This lets us identify those drugs which have the greatest potential to benefit children with neuroblastoma. This allows us to take the most promising drugs forward to clinical trials in children with cancer, often in partnership with The Royal Marsden NHS Foundation Trust.

If you want to find out more about how the ICR’s research using mice is benefiting children with cancer, you can watch the Panorama documentary ‘Can you cure my cancer?’ which features the work of Dr Chesler and the case study of a young girl, Sophie, who is one of Dr Chesler’s patients. Sophie has responded well to treatment with a drug targeting mutations in ALK developed thanks to this work.