Gene discovery could lead to new treatments for childhood cancer

Cancer Research UK

Cancer Research UK-funded scientists have identified two genes linked to Wilms’ tumour – a type of childhood kidney cancer - according to research published in Clinical Cancer Research.

The two genes, called MYCN and FBXW7, provide an important insight into how the disease develops and could lead to new treatments for children with a more aggressive form of Wilms’ tumour.

Wilms’ tumour is the most common form of kidney cancer in children. Each year 80 children are diagnosed with the disease in the UK and around 90 per cent can be successfully treated.

Researchers led by Professor Kathy Pritchard-Jones at The Institute of Cancer Research (ICR) found the two genes by scanning the chromosomes – the structure that contains all of our genetic material – of Wilms’ tumours from nearly 100 children with the disease. They were looking for alterations in the number of copies of thousands of genes that could help them to work out if these genes were linked to a particular type of the disease.

They found that children with a Wilms’ tumour who have an increased number of copies of the gene MYCN are more likely to have high risk forms of the disease1.

Between five and 10 per cent of children with Wilms’ tumour have the high risk form of the disease which is more likely to spread or return after initial treatment and is currently very difficult to treat successfully.

Lead author Professor Kathy Pritchard-Jones, a Cancer Research UK-funded scientist at the ICR, said: “We hope that these findings could open new avenues for treatment and also help target treatments to those children with the more aggressive forms of Wilms’ tumour – the type that’s more likely to return.

“Although treatment for most children with the disease is successful, we now have a useful marker to help us identify those with the high risk form of the disease. Further research is needed before we can use this marker to target stronger treatment to these children, but this is our aim.”

The researchers also found that around four per cent of Wilms’ tumour patients have mistakes in their tumour’s FBXW7 gene. These mutations are associated with a particular type of Wilms’ tumour that is normally successfully treated. This finding could help doctors to tailor treatments to each patient.

It is known that the FBXW7 gene is involved in the breakdown of MYCN, so this new discovery in Wilms’ tumour identifies an exciting new treatment target. There are already treatments in development for neuroblastomas that target the MYCN gene and these same drugs could be used for Wilms’ tumours that return.

Dr Lesley Walker, director of cancer information at Cancer Research UK, said: “Our knowledge of the specific gene defects that cause Wilms’ tumours is limited, we only know the defects for less than half of all tumours, so this work is essential to understand more about the disease.

“This research could be an important step in discovering new treatments for children whose cancer is harder to treat. Cancer Research UK is the largest single funder of children’s cancer research in the country and we’re committed to ensuring more children are cured.”

ENDS

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Notes to Editor

  1. This gene is also linked to aggressive forms of other childhood cancers, especially neuroblastoma, a childhood cancer of the nervous system.