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Hundreds of children with cancer being denied potentially lifesaving drugs

Sarah Knapton
Children would benefit from genetic tests but cannot access precision drugs  - CRUK

Hundreds of children with cancer are being denied potentially lifesaving drugs because youngsters are not included in clinical trials, oncologists have warned.

Recent advances in cancer medicine mean it is now possible to look for specific mutations in tumours which predict how well drugs will work, then choose the most effective treatment. 

In recent years, many drugs have been developed which target such mutations and as well as prolonging life, they can prevent needless chemotherapy and radiotherapy.

But a new study by the Institute of Cancer Research (ICR) found that although 50 per cent of children have cancers which would respond to these precision medicines, just seven per cent have access to the drugs, because they have only been tested on adults.

Researchers said it was ‘deeply frustrating’ that cutting-edge medicines exist that could help children but they cannot be used. They are asking for age restrictions for trials to be lowered from 18 to 12-years-old and for more child-only testing.

Dr Sally George, Consultant Paediatric Oncologist at The Royal Marsden and ICR clinical research fellow, said: “Children deserve the very best cancer treatments, so they can live as long as possible and as well as possible. We desperately need better, more intelligently designed treatments which can give children longer with their families and fewer side effects.

“By testing tumours for specific gene mutations we have shown it’s possible to identify new smarter, kinder treatment options for children, which may potentially give these patients much longer with their families after conventional treatments have failed.

“But our study also exposes the deprerately frustrating barriers that children still face in receiving treatments.

“We know there are regulatory loopholes where companies can avoid testing the drug in children and we need to close those and maybe offer more incentives to companies to get greater access.

“This is the only way we can drive the evidence forward in order to make atual improvements in survival for childhood cancer.”

Researchers looked for 91 mutations in tumours from 233 British children. Some 51 per cent of tumour samples tested had mutations which could be targeted by adult cancer drugs.

Youngsters on the trial with skin cancer who tested positive for BRAF gene mutations were placed on the drugs dabrafenib and trametinib - which are able for children - and all had positive responses for at least nine months after all other drugs had failed. 

Around 1,800 children a year are diagnosed with a solid tumour so the study suggests around 900 could benefit from precision medicine. 

Dr Mike Hubank, Head of Clinical Genomics at The Royal Marsden said: “If you can use a very targeted therapy on a child then it means you are not using a chemotherapy or radiotherapy which can induce more mutations.

“We’re failing future generations of children by not testing and if we don’t start looking for more innovative ways, and that does mean taking a few risks, pharma and regulators are very risk averse. 

“It is understandable that there is a nervousness about testing drugs on children. But there are two kinds of risks, risks now and the risks of not doing something, and I think it’s about getting that balance right and I don’t think we’ve got that balance right at the moment.”

The NHS has committed to offering every child with cancer a genetic test, but until they are able to access the correct drugs, the results are likely to prove fruitless. 

The team also showed that the mutations could be spotted in blood, meaning that the testing could be carried out with a simple blood test rather than an invasive biopsy.

Simon Stevens, NHS chief executive said: “The new era of personalised medicine is well and truly upon us, and this trial shows the life-changing benefits that it can bring for NHS patients. 

“This is exactly why the NHS is to begin offering every child with cancer a genetic test, as part of our Long Term Plan to transform care for millions of people across the country.”

The research was published in the European Journal of Cancer Research.