A Christmas present
no elf can make...

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For some children and young people at Great Ormond Street Hospital, there’s only one thing they want for Christmas this year: a cure...

Every day, research is happening at GOSH to make the future brighter for seriously ill children.

Every family should be together at Christmas. With your support, our researchers can help ensure that more families are able to spend their next Christmas, and the ones after that, at home.

Our Christmas animation stars some very real and very important people, who really understand the life-changing impact of research at GOSH...

At home with Evan

In January 2016, Evan fell over on the way to school. His mum, Clare, became concerned that he wasn’t healing in the right way. He fell again. Within hours of a blood test at his local hospital, Evan was rushed to GOSH with suspected leukaemia.

“The rug was pulled out from under us”, remembers Clare. “We didn’t even realise he was ill. He was always running around, doing drama, playing on his bike or scooter. It was completely and utterly out of the blue. I remember the consultant at our local hospital saying: ‘This is the worst day of your life. It will only get better from here’”.

Over months of tests at GOSH, Evan was dependent on blood and platelet transfusions to keep going. Through a process of elimination, he was diagnosed with Aplastic Anaemia. Stephen, Evan’s father, explains: “The simplest way I always think of it is that Evan’s bone marrow was empty. There weren’t any new blood cells being created by his bone marrow. The doctors at GOSH gave us a longer-term prognosis – at some point, Evan would need a bone marrow transplant.”

For our animation, Evan was illustrated at three different ages.

He underwent chemotherapy to prepare his immune system for the new bone marrow. Evan’s brother, Christian, was in the middle of sitting his GCSEs when he joined his brother at GOSH – ready to donate his bone marrow.

“Christian was allowed in Evan’s room to press the button to start the transplant," says Clare, "then it was strict isolation for four to five weeks until the blood results came back and they’d started to see Evan’s own blood production and his immunity come through again."

The transplant was a success and, after a further six months of isolation back at home, Evan returned to school.

“Evan was absolutely amazing the entire time. He was positive, and very matter-of-fact about it. He kept the nurses amused, he kept us amused – he was singing all the time and constantly putting on shows. He dealt with it far better than we did. He just got on with it, like ‘here’s my medicine, in it goes.’”

“We were lucky with Evan that the ultimate treatment was relatively un-invasive. Christian’s bone marrow cells were hung up in a bag on a drip stand, and just fed into Evan. The doctors said the cells will find their own way into his bone marrow, which they thankfully did. It meant that recovery was quicker so, apart from a single seizure, he wasn’t laid up in bed the whole time - he was quick to recover. For the long term, he had to build back his immunity, but at least his body could focus on that rather than having to worry about surgery wounds or anything like that.”

Clinicians and researchers are striving to provide faster diagnoses and kinder, more effective treatments for children with cancer. GOSH has the biggest children’s cancer unit in the UK and our researchers have helped to dramatically improve survival rates for children in the past 40 years. For Stephen, the importance of research can't be understated. “Without the research into Aplastic Anaemia and Bone Marrow Transplants," he adds," Evan wouldn’t be here now."

‘The beginning of getting better’:
the value of research

Professor Paul Veys is the Director of Blood and Marrow Transplant at GOSH, and treated Evan throughout his stay at the hospital. Every day, he helps drive life-changing care and research in one of the hospital’s most vital disciplines.

Evan was referred to Paul when his bone marrow had become ‘empty’, failing to function as it should. "The bone marrow is the factory for producing all the cells which appear in the blood. Basically, Evan was running out of cells to protect him."

A bone marrow transplant involves finding a matched donor who can provide healthy marrow to replace that which isn’t working in the patient. Luckily for Evan, his brother was a perfect match – but it can be a challenging process to find an appropriate donor, and the operation itself is a highly complex process.

Professor Veys, left, was animated alongside GOSH researchers.

"It’s a bit like sowing seeds of flowers," explains Paul. "You put the seeds back into the patient, into the bloodstream, and the seeds – or stem cells – find their own way back to the garden – the marrow. Then, two or three weeks later, they start to grow back and you see new cells. That’s the beginning of getting better."

Just like the care that children and young people at GOSH receive, Paul knows how the research process cannot stop over the festive season. "Many of the patients who are in the wards having transplant procedures are on research protocols or are receiving research products, so pretty much researchers are on duty, constantly thinking 24-hours round the clock. Patients are supervised constantly – it never stops."

"The research teams and the clinical teams at GOSH are entwined,’ says Paul, ‘It’s a two-way language, a conversation. We tell our researchers what our problems are, what we’re not achieving in the clinical setting, and then they can come up with solutions. Likewise, they can tell us the things they’ve found in the research laboratories, and we can immediately interpret that into what might benefit the child."

Professor Veys has been working with transplants at GOSH since the 1990s, during which time great progress has been made. However, he believes there are still two key areas for progress that research can unlock: making the process safer, and improving results for donors that are not ideally matched. "Research is vital to improving results" says Paul. "We are at the cutting edge of changing treatments, and many things that we can change or improve here are immediately disseminated around the world. The rest of the world is looking for us to find answers where currently none exist."

The difference you make

In the last year, GOSH Charity has provided more than £13.6 million for research projects – at GOSH and across the country. Paul is clear about the critical difference that charitable support makes. ‘The money people raise for GOSH goes into research that makes a difference fast. Due to the close ties between researchers and clinicians at GOSH, we can move research results into the clinical environment extremely quickly. So charitable support is incredibly important – I think that if the children could thank our supporters themselves, they would do.’

‘A successful transplant will return a child to near ‘normal’ life. Failure can mean that, sadly, we will lose the child. These donations have made a difference to many lives already, and it is lives we’re talking about here. I think if donors could see the children, it would be very warming for them to see the results that their fundraising has already produced.’