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Thinking outside the box to tackle an aggressive children’s cancer

by Joanna Lewin | Interview

8 September 2021

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Dr Sam Behjati
Dr Sam Behjati

From the body parts affected and the treatments employed, to the long-term side effects experienced, children’s cancers differ fundamentally from adult cancers.

While the latter most commonly arise from decades of acquiring mutations each time a cell divides, the causes of children’s and young people’s cancers are less well understood, although we believe that many are due to errors that occur during embryonic development. As a result, some cancers that occur in children and young people almost never emerge in adults and vice versa.

The need for distinct approaches and unique research is clear. That’s why, back in March, we joined forces with fellow charity Children with Cancer UK to co-fund the Cancer Research UK–Children with Cancer UK Innovation Awards, granting 5 teams up to £1m each to realise creative, ambitious projects that we believe can bring about game-changing discoveries.

Six months on, we’re back on a video call with Dr Sam Behjati, one of the team’s co-leaders, to hear how the funding is allowing his team to use big data to chart the origins of a rare children’s cancer called rhabdomyosarcoma.

“We must try something new”

Rhabdomyosarcoma is believed to originate in cells that play an important role in embryonic development.

Around 55 children are diagnosed with the disease each year in the UK. For some children, if they have a certain sub-type of rhabdomyosarcoma and it’s caught at a very early stage, the disease can be effectively treated. “However, most children with rhabdomyosarcoma do not fall into this category,” explains Behjati, who’s based at the Wellcome Sanger Institute and co-leads the award-winning team with Dr Karin Straathof at University College London. “Unfortunately, many children die from the disease.”

What’s more, progress has been slower for this cancer type than other children’s and young people’s cancers. Thanks to long-term investment in research, we’ve come a long way since the 1970s when children’s cancer survival was much lower than it is today. Now, on average, around 8 in 10 children and young people diagnosed with cancer will survive their cancer for 10 years or more.

“All cancers are horrible, but some of them we’re doing okay at treating,” explains Behjati. “But in rhabdomyosarcoma, we’ve hit a brick wall. We’ve tried chemotherapy in all its combinations, variations and intensities, and we’re still seeing very poor outcomes. The status quo is unacceptable. We must try something new.”

An opportunity to think big

Enter the Cancer Research UK–Children with Cancer UK Innovation Awards, which give researchers the funding and creative freedom to explore new avenues of investigation, in the belief that they could drastically improve outcomes for children and young people facing cancer in future.

“That the funders had the vision to support blue-sky thinking is astonishing – it’s exactly what we need to tackle this horrible disease”

“What distinguishes these awards is that they’re so much bolder in approach and ambition,” says Behjati. “That the funders had the vision to support blue-sky thinking is astonishing – it’s exactly what we need to tackle this horrible disease.”

And blue-sky is fitting, given Behjati’s team – which he describes as a “brilliant hodgepodge of people” – contains an astrophysicist, alongside a mathematician, a bioinformatician, clinicians and PhD students “from all strands of research life”.

Together, they’re pooling their varied skills and expertise to collect and analyse large amounts of genetic data that will give us greater understanding of how rhabdomyosarcoma begins and develops – information that could be used to develop new, more effective treatments with fewer side effects.

Creating a cell ‘atlas’ to find new treatment targets

Behjati and his team are working with clinicians at Great Ormond Street Hospital – the UK’s largest paediatric oncology unit – to collect tumour samples from children with rhabdomyosarcoma in their care.

The team then apply single-cell RNA sequencing – a process that allows Behjati and his team to ‘read’ every individual molecule and cell within each sample. “This technology shows us all of the cancer and non-cancer cells in the sample and tells us what they do,” explains Behjati. “So, you learn what it means to be a rhabdomyosarcoma cancer cell but also about how it interacts with healthy cells.”

By creating an intricate cell ‘atlas’ to identify and lay out every component of a sample, and comparing and measuring this against a healthy equivalent, the team hopes to spot the differences and find potential treatment targets.

And they’ve already made some interesting findings, including the discovery of foetal genes that are repurposed by rhabdomyosarcoma cells. These genes shouldn’t exist after birth – they only persist to do cancer’s bidding. “These genes are highly attractive to us because we could target them with treatments,” says Behjati. “This discovery opens up a whole other box of potential translational applications of the data that we generate.”

For Behjati and his team, who are still only at the beginning of this exciting project, the future already looks brighter for children with rhabdomyosarcoma.

“It’s lovely to see some progress,” he says, “I’m very hopeful.”