Discovering better treatments for children with brain tumours

A few months after developing devastating headaches, five-year-old Jasper Lilley was diagnosed with a type of brain tumour called medulloblastoma. After surgery to remove the tumour, he went through a year of intensive radiotherapy and chemotherapy. It was, his mother Alice remembers, “horrendous”.

As well as funding the Children’s Brain Tumour Centre of Excellence, Cancer Research UK was the catalyst for this whole endeavour.

– Professor Raj Chopra, Drug discovery expert

“He was vomiting, he was throwing up loads. It was horrible. I felt helpless. I thought ‘I just want to take him home’.”

“I literally got to the point of thinking ‘He’s not going to make it through the chemo because he can’t keep anything down’. And even weeks after the first cycle he was still reacting really badly to the chemo. And then six weeks later, he had to go back in again for his next round. And we had to keep going on like that for nearly a year.”

Thankfully, Jasper’s now back to his usual cheeky self – his hair’s grown back, he’s back at school, and he regularly goes swimming and plays rugby with his dad at weekends. Life is back to normal. But the experience has changed the family’s outlook forever, and Jasper is taking growth hormones as a result of the long-term side effects of his treatment.

£3 million for children’s brain tumour research

At Cancer Research UK, we want to develop the next generation of treatments for children with brain tumours. Treatments that are better, and cause fewer of the life-changing side effects that hit Jasper so hard.

But to do this, we need to revolutionise our understanding of what makes these cancers tick. And so, this year, we opened the Cancer Research UK Children’s Brain Tumour Centre of Excellence – a £3 million investment that aims to accelerate progress in developing and testing new, better and kinder treatments for children and young people with brain tumours.

Professor Raj Chopra works at The Institute of Cancer Research in London, one of two world-class research groups behind the new Centre.

“This new Centre of Excellence is very much a partnership between the Cancer Research UK Cambridge Centre, led by Professor Richard Gilbertson, and several of us here at The Institute of Cancer Research,” he says.

Speeding up progress

According to Gilbertson, funding for research into new treatments for children with brain tumours has been limited until now.

“The problem is, most cancer therapies are not developed specifically for children,” he says. “Children’s brain tumours are rare so pharmaceutical companies tend not to develop drugs specifically for children. Rather they will do this for more common cancers, including adult brain tumours.”

But children’s brain tumours are biologically different to adult brain tumours.

“You can’t always just take drugs off the shelf that have been developed for adult brain tumours and expect them to work in children,” Gilbertson adds. “You can’t set out with that assumption.”

That’s why Gilbertson believes the new Centre is so important.

“We’re no longer going to beg, borrow and steal drugs from adult cancers. This new Centre aims from the start to design new drugs specifically to treat children’s disease.”

This new Centre aims from the start to design new drugs specifically to treat children’s disease.

– Professor Richard Gilbertson, Children’s cancer expert

Why this Centre is different

Chopra describes the new Centre as a partnership between biologists and chemists.

“The exciting thing about this is it’s the full circle: the biologists develop new, more accurate ways to study brain tumours and to understand their biology, and find possible targets for drugs.

“Then the chemists find drugs where we really understand the chemistry of how they work, and go and test out whether they work against the targets the biologists have identified. It’s testing if the chemistry actually affects and targets the biology in the way we expect it to.

“One thing we’re focusing on and are very excited about in particular is that, over the years, we’ve developed ways to destroy particular proteins inside cancer cells, allowing us to manipulate how the cells grow. We’re now going to be using this technique to try to better understand exactly how brain tumours develop, and how we can develop new drugs to kill them.”

The team plan to use an array of sophisticated techniques to study how brain tumours work and to identify and assess promising new experimental drugs, which can then be tested in rigorous clinical trials to see if they’ll be of benefit to patients.

“When Richard was working in the US, his team there did initial tests looking at about 1.2 million compounds to see if they killed brain tumour cells in the lab. Now, we’re going to be working with him to see exactly how these compounds work to kill brain tumour cells, and if any of them can be developed into proper drugs.”

Our impact

It’s vital, cutting-edge work, none of which would be possible without support from Cancer Research UK, says Chopra.

“As well as funding the Children’s Brain Tumour Centre of Excellence, Cancer Research UK was the catalyst for this whole endeavour,” he says. “They’ve brought together the brain tumour expertise of two Institutes that really complement each other. There’s a huge amount of excitement about it, and it’s already given us a platform to develop more collaborations with researchers in other countries.”

Plans on how to spend the £3 million are already being put into action. The team has started recruiting scientists to join two new research groups, dedicated to finding new treatments for children’s brain tumours. Measures have also been put in place to ensure that crucial infrastructure is laid out to help bring the brightest minds in children’s brain tumours together.

“The science is obviously important,” says Gilbertson, “but unless you have the tools, the people and the organisation to actually assess those ideas we’re not going to go anywhere”.

He says it’s a bit like having the best racing car driver in the world with no car to drive.

“Cancer Research UK is providing us with the racing car, so we can put the drivers in it. That kind of funding is not common and that’s why the Centre is so important.”

It’s urgent work: around 410 children develop brain tumours every year in the UK, most of whom will have intensive therapies to treat their disease. Many experience long-term side effects. And while the majority will be cured, these cancers claims the lives of about 80 children every year. New treatments can’t come soon enough – and thanks to the new Centre, they surely will.

Read more about the progress we’ve made in the last 12 months in our Annual Review 2017/18.