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Great Ormond Street Hospital treatment clears girl’s incurable cancer | UK | News



A teenager’s family are on “a strange cloud nine” after her incurable cancer was cleared from her body in the first use of a revolutionary type of treatment. Thirteen-year-old Alyssa was diagnosed with T-cell acute lymphoblastic leukaemia in 2021 and underwent all the current conventional therapies, including chemotherapy and a bone marrow transplant.

But her disease came back and there were no other options available apart from palliative care.

So in May this year, she and her family decided she would be the first person in the world to try an experimental treatment for her leukaemia.

She became the first reported patient in the world to receive base-edited T-cells at Great Ormond Street Hospital for Children (GOSH), in collaboration with the UCL Great Ormond Street Institute of Child Health (UCL GOS ICH), to treat her “incurable” T cell leukaemia.

Alyssa said: “Once I do it, people will know what they need to do, one way or another, so doing this will help people – of course I’m going to do it.”

She was admitted to the bone marrow transplant unit (BMT) at Great Ormond Street Hospital (GOSH) to receive to receive genetically modified CAR T-cells that originally came from a healthy donor.

These cells had been edited by scientists using new base-editing technology to allow them to hunt down and kill the cancerous T-cells without attacking each other.

Just 28 days later, she was in remission and went on the receive a second bone marrow transplant (BMT) to restore her immune system.

Now, six-months post-BMT, she is doing well at home recovering with her family and continues her post-BMT follow-up at GOSH.

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Alyssa is the first patient to receive this treatment during the trial but the team are aiming to recruit up to 10 patients with T-cell leukaemia who have exhausted all other treatment options.

This trial is only accepting patients eligible for NHS care. Any patients eligible to receive treatment under the NHS and interested in this trial should approach their specialist healthcare provider.

The Bone Marrow Transplant (BMT) and CAR T-cell therapy teams at GOSH hope that, if the trial is successful, the treatment could be offered to children earlier in their treatment journey.

They are also hoping that the technique could be an option for other types of leukaemia.

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