Kinder cancer treatment tested at Great Ormond Street Hospital

Clinicians at Great Ormond Street Hospital (GOSH) led a study of Blinatumomab treatment for children diagnosed with B-Acute Lymphoblastic Leukaemia (B-ALL) who have incredibly bad side effects. It was tested at GOSH and over 20 centres across the UK and Ireland.

Blinatumomab, which is already used in children and adults for resistant B-ALL, teaches the immune system to target cancerous leukaemia cells. It is hoped it could initially help around 40 children and young people each year.

Arthur, 11, was one of the patients who tried the new treatment at GOSH.

Mum Sandrine said: “It came after two rounds of chemotherapy, and we had slowly slipped into Arthur being more withdrawn, sleeping more and becoming weaker, alternating with the periods on steroids where he would be ravenous and hyperactive. It was completely out of his control, we were living in a constant challenge as his body was getting hit by the drugs.

“We were curing him by making him feel worse, it’s a very difficult thing to process.

“Switching to the new treatment didn’t actually seem that difficult a choice, we felt very, very fortunate that were able to go down the antibody route.”

The family had been warned there could be an ‘inflammatory period’ of fever as Arthur got used to the new treatment. He had to spend 10 days in hospital. But once the treatment settled, they noticed a difference, his mood brightened, his appetite stabilised and his hair grew back, which felt like a recovery.

Sandrine said: “At the end of the Blina treatment we had a nice outing, we planned a picnic, we climbed trees in the woods, went for a walk. We wanted to enjoy this time before Arthur had to go back on the next phases of the treatment. The final maintenance phase of chemotherapy eroded exercise, and we had to stop anything that was too tiring.”

Dad René said: “Blinatumomab was like a little bit of sunshine. It was the first part of the treatment that really seemed to work, and that makes a big difference.”

Arthur had to return to hospital every four days during the immunotherapy but was able to carry a small backpack the rest of the time which allowed the treatment to continue.

Sandrine said: “He was able to have the medicine with him rather than being in hospital, he enjoyed the fact that he was able to hold it and be responsible. He embraced all of it.”

Arthur has his final round of chemotherapy in December 2022, and at the end of April he had the final operation to remove his port, which allowed treatment to be given and blood to be taken.

Sandrine said: “It was a big step, he was free.

“Since mid-March he has been on an upward slope, he’s gone from regularly missing school to great attendance, and being able to do one or two activities after school.

“He’s doing fantastic and we’re so grateful for continued research into better cancer treatments, and the wonderful care Arthur has received.”

Two-year-old Noah was diagnosed with leukaemia in May 2022 and started treatment straightaway. Unfortunately, he ran into complications, which caused a stroke.

His mum Dilly said: “He was bedridden for best part of two months with the first four weeks he could hardly move. He had stopped talking, walking and eating but Blinatumomab made such a difference as he started to recover from his stroke. He started making facial expressions, some words and even walking so to give him invasive treatment for leukaemia could have set him back.

“His backpack carrying the Blinatumomab was ok, it took some getting used to especially as he was just learning to walk again. Changing the cassette in the backpack every four days was ok and relatively quick, if you live far from GOSH then I can imagine that will be difficult.

"We didn’t see any side effects and Noah continues to improve and eventually he was actually able to wear the backpack and carry it himself.”

Consultant Paediatric Haematologist Sujith Samarasinghe was one of the leads on the study, which has so far seen 105 patients given Blinatumomab instead of two blocks of chemotherapy, over an eight-to-10-week period.

He said: “This is a game-changer in the search for kinder cancer treatments. We found Blinatumomab can replace the most intense, nasty blocks of chemotherapy, and get rid of the leukaemia without causing awful side effects.

“And the most amazing thing is the children have gone back to some degree of normal life, as they’re able to focus on being themselves again.”

Ajay Vora, Consultant Paediatric Haematologist at GOSH, who also led the study said: “This study has shown the treatment can work and will hopefully be the platform to see how we can use it to help more children.

“It’s important we continually push ourselves to find not only the most effective but also the kindest treatments.”

UK guidelines mean only patients who meet certain criteria will be eligible for the treatment. Patients and families will need to ask their clinician, who will check if they meet the criteria, but should be aware it may not be available.

Clinicians hope to test start clinical trials on this treatment over the course of the next few years to see if it can be used for all patients with B-ALL.

Replacing outdated buildings on Great Ormond Street, our new Children’s Cancer Centre will mean that children with rare and complex cancers will receive care in the best possible environment, making it easier for them to be able to play, continue with school and participate in normal activities. The CCC will also enhance our ability to research and innovate to develop new and kinder treatments for cancer.

With significant outside space, a new hospital school, child centre inpatient wards and day care spaces, imaging, theatres and critical care services linked to the existing hospital, children and young people coming to GOSH will have access to the very latest technologies and receive care and treatment in environments that reflect their needs.

For more information visit Children’s Cancer Centre.

GOSH Charity is fundraising for the Children’s Cancer Centre, find out more about how you can be part of Build it Beat it.