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Infant saved by genetically modified T cells - first treatment

Friday November 6th, 2015

An infant appears to have been cured of advanced leukaemia using an experimental treatment with genetically modified T-cells, it was announced yesterday.

The child, now aged 16 months, became the first patient to try the treatment, which was under development at Great Ormond Street Hospital, London, UK, and University College London.

Doctors gained special ethics permission to test the use of the UCART19 cells after all other treatment options ran out for the child.

The cells have been designed to be invisible to leukaemia drugs and to target leukaemia cells. Until now they have only been tested in laboratory mice.

The patient, Layla, was suffering from relapsed acute lymphoblastic leukaemia.

Layla had undergone chemotherapy, bone marrow transplants and another experimental treatment in Sheffield. Her family had been told that no further treatment existed.

Two weeks after a 1ml infusion of the UCART19 cells, Layla developed a rash, suggesting a working immune reaction.

After two months, Layla was free of cancer and received another bone marrow transplant.

Her father Ashleigh said: "Even though she is well at the moment, we still don’t know what the future holds. She will still have monthly bone marrow checks for now and might be on some medicines for the rest of her life."

Consultant immunologist Professor Waseem Qasim said: "We have only used this treatment on one very strong little girl, and we have to be cautious about claiming that this will be a suitable treatment option for all children.

"But, this is a landmark in the use of new gene engineering technology and the effects for this child have been staggering.

"If replicated, it could represent a huge step forward in treating leukaemia and other cancers."

Professor Paul Veys, the hospital's director of bone marrow transplant, added: "As this was the first time that the treatment had been used, we didn’t know if or when it would work and so we were over the moon when it did.

"Her leukaemia was so aggressive that such a response is almost a miracle."

Tags: Cancer | Child Health | Genetics | NHS | UK News

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