Genetic test to enable personalised cancer medicine for children

31 March 2016

It has been announced that a pilot trial of a new genetic test for children with cancer will take place across the UK.

The test involves sequencing a panel of 81 different genes from tumour DNA samples, with a view to informing the choice of biologically targeted therapy for each child and tumour. The hope is that this more personalised approach to initial therapy will produce positive results, both in terms of attacking the tumour and minimising harmful side effects. It was developed at the NIHR Biomedical Research Centre at The Royal Marsden and The Institute of Cancer Research (ICR), with funding from the charity Christopher’s Smile.

A total of 400 or so children under the age of 14 with solid tumours will be enrolled in the trial via specialist centres at 21 different hospitals across the UK, including the Royal Marsden NHS Foundation Trust in London. If the results are good, the trial will be expanded and may eventually make the test available to all children with solid tumours in the UK. Solid tumours are typically somewhat more difficult to treat effectively than forms of blood cancer among children.

Although the initial pilot is small, this is an important development in a number of respects. It should help to identify the most common genetic changes that drive typical childhood cancers, and thus identify the most appropriate treatments currently available. It may also help to drive the development of new treatments of this kind. Whilst these approaches are becoming an increasingly common form of personalised medicine among adults, they have not been so widely available to paediatric cancer patients – partly because it is more difficult to set up clinical trials involving children, and hence to determine how effective different treatments are.

Karen Capel, who founded Christopher’s Smile with her husband following the death of their young son from a brain tumour, said“There is an urgent unmet need to provide new treatments for those children diagnosed with the most aggressive and hard to treat cancers. We believe this gene sequencing test is the key ‘foundation stone’ in enabling personalised medicine for children. It will help to bring new treatments for children a step closer”.

More targeted cancer treatment is in fact particularly important for children, not only to improve survival rates but also because the long-term impact of treatment side-effects in young people who do survive can be particularly severe. Christopher’s Smile says that the charity’s vision ‘is for every child diagnosed with childhood cancer to not only survive, but to reach adulthood enjoying a good quality of life’ .

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