Cancer treatments ‘not being trialled in children’

Grace died when she was four years oldTrialling more cancer drugs on children could help children like Grace in the future, her mum says

Children with cancer could be missing out on potentially life-saving drugs because EU regulations allow drug companies to opt out of running trials in children, cancer experts say.

Since 2012, 62% of approved EU cancer drugs were not tested on under-18s.

But cancer scientists say the rules should be changed so that adult cancer drugs have to be tested on children.

They are also calling for the age limit for adult clinical trials to be lowered to include teenagers.

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The Institute of Cancer Research in London, cancer hospital the Royal Marsden and parents of children who have died from cancer say nowhere near enough cancer medicines are being trialled in children or licensed for use in children.

A consultation is currently running on the future of EU rules on clinical trials, which means there is a chance they could be changed.

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What happened to Grace?

Grace Kelly was four years old when she died from a very aggressive tumour in her kidney, just three weeks after she had been diagnosed, in 2014.

Her mother, Jennifer, who is a GP, believes the regulations should be tightened to allow more tests of cancer drugs on children to take place.

“There could have been a treatment for her out there, but we were told at her diagnosis that her cancer was [terminal],” she says.

She says huge progress could be made in treating rare cancers if the rules were changed.

“They are not looking at the children right now,” she says.

The Grace Kelly Ladybird Trust has been set up in her memory to push for better treatments for children with cancer.

Grace KellyImage Grace had started school a month before she was diagnosed with cancer
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Why can drug companies opt out of trials in children?

The rules, called the EU Paediatric Regulation, which were set up in 2007, allow drug companies to be granted a waiver by the European Medicines Agency (EMA) when it comes to trialling cancer drugs on children.

This usually happens if the drug in question targets a cancer such as lung cancer that does not affect or is very rare in children.

But cancer researchers and charities say the way a drug works may mean it could be effective in children – even if it does not seem to be relevant.

Cancer medicines are changing, and they are often designed to treat many different types of cancer, which may have the same genetic target, rather than just one.

So they want fewer waivers to be issued and more trials on children to take place.

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Why do drug companies want to avoid carrying out trials in children?

It is not that they do not want to carry out the trials and help cure childhood cancers – it is more that they want to make sure the trials are worthwhile.

Not all cancer drugs will be relevant or safe for children.

Creating cancer drugs is an expensive process and developing treatments for children, in whom cancers are rare, is a financial challenge for many manufacturers.

That is why it has been suggested that greater economic incentives are needed for companies to run trials in children.

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Are drug trials on children really complex?

Doctors have to be very careful when treating children with cancer, of course.

They will always follow up closely after drugs are given – but children with cancer are capable of being treated just like adults, often with the same drug doses.

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What difference could tightening the rules make?

Prof Paul Workman, from the Institute of Cancer Research, says children are missing out on innovative cancer treatments now common in adults.

As a result, he wants the regulations to be updated so that children with cancer – even if it is rare or terminal – get the best possible treatment with the latest medicines.

Prof Louis Chesler, honorary consultant in paediatric oncology at the Royal Marsden NHS Foundation Trusts, says access to cancer medicines has improved in recent years and more cancer drugs are being developed – but there are still improvements that could be made.

“Is there any reason why adult cancer trials can’t lower their age limits to 16, or even 12?” he asks.

And he adds: “Everyone realises this is a challenge, but more incentives are needed for drug companies to make a return and to speed up the development of drugs.”

New Frontiers in Cancer Treatments


For decades, surgery, radiation and chemotherapy have been the three staple treatments against cancer. But clinical trials over the past five years have shown that harnessing the body’s immune response—which evolved to fend off harmful bacteria and viruses, among other things—presents a new treatment alternative. In fact, for some types of cancer, clinical trials of immunotherapy have reported complete remission in 90 percent of cases. The April issue of Scientific American highlights these emerging therapies, but we have covered the growing promise of immunotherapy for quite a while. In this special digital package, we have pulled together other recent Scientific American articles about different types of immunotherapy that tell you even more about this exciting next generation of cancer treatment.


The Cancer Defense

Against cancer, new enhancements to the body’s own immune system are looking like lifesavers

By Karen Weintraub on April 1, 2016

What Gene Therapy Needs Now: A Good Off Switch

Researchers are developing molecular switches that can inactivate transplanted genes, paving the way for safer gene therapies. First up—immunotherapies for cancer

By Jim Kozubek on January 1, 2016

Can Viruses Treat Cancer?

For some cancer patients, viruses engineered to zero in on tumor cells work like a wonder drug. The task now is to build on this success

By Douglas J. Mahoney, David F. Stojdl, Gordon Laird on November 1, 2014

New Drugs Free the Immune System to Fight Cancer

By releasing the brakes that tumor cells place on the immune system, researchers are developing a new generation of more powerful treatments against malignancy

By Jedd D. Wolchok on May 1, 2014

A New Ally against Cancer: Vaccines

The FDA recently okayed the first therapeutic cancer vaccine, and other drugs that enlist the immune system against tumors are under study

Starving cells of sugar could be the key to future cancer treatments

Here’s what happens when you take away a cancer cell’s fuel.

Cancer cells are cells that cheat death – rather than growing old and dying, as healthy cells do, they continue to grow and divide, changing their metabolism to continue proliferating throughout the body. It’s that proliferation of malignant cells that gives rise to cancers and tumours, and the challenge for medical researchers is to prevent this abnormal behaviour from occurring.

One way cancer cells fuel their growth and expansion is by increasing their glucose intake, and researchers are now looking at the possibility of shutting this down to combat their spread. A recent study has identified the PARP 14 protein as being responsible for the additional glucose production, and it’s overproduced in virtually every human cancer, but isn’t found in normal, healthy cells.

Genetic and molecular tests have shown that reducing PARP 14 levels in cancer cells literally starves them to death, as the UK-based team describes over at The Conversation. What’s more, scientists have discovered that the level of PARP 14 also influences the chance of patients surviving cancer, and this could help doctors identify the aggressiveness of a particular cancer ahead of time and adjust the treatment accordingly.

While a PARP 14-inhibiting drug is still a long way from becoming a reality at the moment, the findings have the potential to make a huge difference in cancer treatments once the necessary development and testing has been completed. Unlike chemotherapy and radiotherapy techniques, a PARP 14 blocker would only affect the cancerous cells, so treatment would be much kinder to the patient’s body.

The authors of the study, published in Nature Communications, believe that processes like chemotherapy would still be necessary, but a drug that targets the protein that cancerous cells rely on would make these cells much more vulnerable to chemo – as a result, doses could be lowered or programs could be shortened.

Glucose is so important to cancer cells because it not only gives them energy, it also guards against the molecules that cause natural cell death. If that glucose is removed, the cancer cells have their guard down. But it’s not quite as simple as cutting out sugar from your diet, because cancer cells adapt to find alternative fuel sources to build up glucose levels.

Researchers need to first design a drug that blocks or reduces PARP 14, and then make sure it’s safe and effective for humans, but our understanding of how cancer spreads – and how it can be stopped – is growing all the time, and the PARP 14 discovery could be one of the most important finds yet. “If we can find a way of stopping this over production of PARP 14 we can cure cancer,” says one of the team, Salvatore Papa from the University of London.