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Newcastle Children’s Bone Marrow Transplant Unit discover new treatment

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Newcastle’s Children’s Bone Marrow Transplant Unit have discovered a pioneering new treatment to tackle complications that can arise from stem cell transplants.

The new procedure will tackle GVHD, an often fatal complication of stem cell transplantation, when the transplanted cells attack the patient.

The breakthrough came after all typical treatments for GVHD on a child who had a transplant for Severe Combined Immunodeficiency (SCID), had failed. SCID is a condition where children are basically born without an immune system. The condition is fatal and the only effective treatment is a stem cell transplant.

4-year-old Kristina Vukolova Credit: Bubble Foundation

Four year old Kristina Vukolova, from County Cork in Ireland, became the subject of the ground-breaking new procedure. The new treatment uses the patient’s mother’s stem cells even though she was only a half identical tissue type match.

She stayed in what is known as ‘The Bubble Unit’ at the hospital, which uses a sterile ‘bubble’ to protect children from any bugs and bacteria while they are undergoing treatment.

Mum, Jekaterina, 29, spoke about her experiences:

“When Kristina was six months old, we knew

something was very, very wrong. After being diagnosed with SCID, she was admitted to Newcastle’s

Children’s Bone Marrow Transplant Unit where a donor was found and doctors moved quickly to perform

her first transplant.

“When the doctors said performing this new type of treatment was an option, I was happy to go for it as I

knew they’d tried everything else.

“Kristina did well through the chemotherapy she needed to prepare her for the transplant. At this point she

was very poorly though and I was very worried. Incredibly, as I was used as the donor and only a 50 per

cent match, the doctors were able to take some of the T cells out and give her the ones that she needed.

It’s the first time this treatment has been performed for this specific condition.”

– Jekaterina Vukolova
Kristina and her mother Jekaterina Credit: Bubble Foundation

Research at Newcastle’s Children’s Bone Marrow Transplant Unit is supported by charity, The Bubble Foundation, which helps fund the research needed.

Since the charity was founded, survival rates have increased from 50 per cent to around 90 per cent.

Professor Andrew Cant, a world-leading pediatric consultant, said children with immune deficiencies are often a forgotten group:

“We need funding through The Bubble Foundation to help research conditions like Kristina’s further still. Children with Primary Immune Deficiencies are often a rather forgotten group, even though around one in every 500 babies is born with some form of immune deficiency. It’s my vision that all children with this condition will be diagnosed as small babies, we’ll have treatment that is 100 per cent successful, 100 per cent of the time; less toxic and less demanding than it is today.

We’ve come a long way, but there’s still a huge amount to do.

– Professor Andrew Cant