What’s it like for your child to be part of a clinical trial?

We’ve funded many of the world’s most successful clinical trials into children’s and young people’s cancer treatments. These trials are the crucial final step in turning lab science into medicines that help children and young people live longer, better lives beyond cancer.  

In fact, it’s largely thanks to clinical trials that more than 8 in 10 children and young people (aged 0-24) diagnosed with cancer in the UK today will survive for at least 10 years. Differences in the statistics make an exact comparison difficult, but that number has roughly doubled since the 1970s. Our role in that story is shown by the fact that, today, 9 in every 10 children and young people treated with cancer drugs on the NHS receive a drug linked to our work. 

As far as we’ve come, we know there’s more work to do. We also know we won’t be doing any of it alone. Our trials involve scientists, doctors, nurses and a whole host of specialists, but they couldn’t happen at all if people affected by cancer weren’t willing to take part. Since 2002, around 18,000 children and young people have done just that, each with a family or support system behind them. 

But what’s it like to be that support system? Caring for a child or young person with cancer is never easy, even when they’re being treated with drugs that have already been through clinical trials. No parent plans for their child to start shaping the future of medicine before they turn 25. How does it feel when they do? 

Here, five parents share their experiences of five of our clinical trials. 

“She was so inspired by the nurses” – Aleesha-Marie and MyeChild01

Aleesha-Marie was invited to join our MyeChild01 trial after she was diagnosed with acute myeloid leukaemia (AML) in October 2019.  

“She was 13 at the time,” says her mum, Emma. “We asked her if she wanted to be on the trial, but we said that it was her choice and she didn’t have to.  

“Without the MyeChild trial, Aleesha-Marie would not have had her transplant so early and her outcome most definitely would have been very different,” says Emma. “As well as the treatment, there is also extra follow-up which has been very reassuring.” 

Emma says treatment was a tough experience for all the family.  

“It was so hard signing the forms for all the treatments – not just the trial. I remember the very first one we had to sign about having a line fitted and a bone marrow aspiration. We were so scared she might not make it.  

“We didn’t hesitate” – Luke and SIOPEL-6

Clinical trials can also test ways of reducing the side effects of existing treatments. SIOPEL-6 looked at a new drug that could be used to reduce hearing loss in children treated with a specific type of chemotherapy for the rare liver cancer hepatoblastoma.  

Luke was diagnosed with hepatoblastoma in December 2007, when he was just six months old. He started treatment at Great Ormond Street Hospital for Children in London on Boxing Day.  

“Luke started with four rounds of fortnightly chemotherapy, followed by a seven-hour operation and more chemotherapy,” says Luke’s mum, Claire. 

“If he had suffered hearing loss at that age, it would have had a huge impact on his long-term quality of life. We didn’t hesitate to take part in the trial, for Luke and for other children in the future.  

“We felt we were well looked after on the trial, and Luke had lots of extra hearing checks. The outcome has been as hoped. A recent hearing check confirmed he has normal hearing in both ears.  

“His treatment finished in April 2008. He’s 17 now, so he has just had his last check-up at GOSH and will now be looked after by the team at University College London.  

“He loves gymnastics now and competes nationally – he was in the British Championships in Liverpool in March.  

“We need to minimise the long-term health impact on children and find treatments that are kinder but still effective.” 

“The hardest thing was to balance our minds” – Faye and FaR-RMS

Not everyone who takes part in clinical trials receives a new treatment option. Randomised trials are divided into experimental arms, where patients receive the treatments that are being evaluated, and control arms, where patients receive the standard treatment they would have if they weren’t in the trial. This is so researchers can compare how well different options work. 

Six-year-old Faye from Liverpool was part of two control arms of the FaR-RMS trial, which is testing multiple ways of improving rhabdomyosarcoma treatment. 

“It’s quite hard to imagine the therapy or the machine if you haven’t seen it in action but it was like boarding a spaceship,” Bex explains. “Faye had to be completely alone in the treatment room but the play specialists and radiography teams worked so well with her that she actually really enjoyed it – they used to play her favourite songs from The Lego Movie soundtrack while she had treatment. 

“She suffered quite badly with side effects like skin burns and tiredness but it didn’t stop her excitement. On every photograph she’s beaming smiles. 

“After that radiotherapy finished, Faye had maintenance chemo for six months from September to early March. For our second part of the trial, the randomisation was not done until about a month from the end of the standard schedule, so it was difficult as we were not sure if we were preparing for an extra six months of treatment or not. 

“That was the hardest thing about the trial, to balance our minds and to prepare Faye for what was happening.  

“We needed to keep thinking of the positives of either scenario. If we did have the extended dosage, it would be good to be having more treatment which we knew was working. But if we finished earlier, then it would be good to finish earlier and get back to ‘normal’ life!  

“The uncertainty was difficult with my work too. I had taken time off after Faye was diagnosed and I wasn’t sure when treatment would finish and if I could go back. We just wanted to settle our minds, but we also understood that this was part of the trial. 

“There were no other options” – Abdullah and BEACON

Clinical trials can be especially valuable for young patients when standard therapies aren’t working, or if their cancers come back and are no longer treatable with the same drugs. 

Bushra, whose son Abdullah was diagnosed with neuroblastoma in 2017, says that the BEACON trial was her family’s “last hope”.  

They were invited to take part in the trial after two full courses of chemotherapy had failed to stop Abdullah’s tumour from growing.  

BEACON was set up to find a better treatment option for children and young people in this position. “There were no other options so we signed up thinking that, even if it didn’t benefit Abdullah, it might help someone else,” says Bushra.   

Abdullah was given a new treatment combination of chemotherapy and bevacizumab. Bevacizumab is designed to stop tumours from creating blood vessels, limiting their ability to bring in the food and oxygen they need to survive and grow.  

In a matter of months, the treatment shrunk Abdullah’s tumour by more than half, meaning he could have a life saving stem cell transplant.  

By January 2019, he was fit enough for surgery to remove what remained of the tumour. He then had 10 weeks of radiotherapy.  

“Abdullah has always been very strong and positive, but he does remember treatment,” says Bushra. “He is now back at school and is mad about football.” 

“It gives me hope” – Alayna, rEECur, and the work ahead

Like Abdullah, Vea’s daughter Alayna joined a clinical trial because there weren’t any other standard treatment options left. She had been diagnosed with Ewing sarcoma in 2012, when she was 10. Although the first treatment was successful, Alayna’s cancer returned in 2018.  

At that point, she joined the rEECur trial, which was testing how well different types of chemotherapy work for Ewing sarcoma when standard treatment can’t stop it from growing, or if it returns. 

“Trials are so amazing,” says Vea. “And Alayna did well as part of rEECur – her results at the end of the trial were good, and she finished radiotherapy just in time to collect her GCSE results.”