Teenager is ‘leukemia-free’ after breakthrough cell-editing treatment | Scientific and technical news

A 13-year-old boy has become cured of leukemia thanks to a revolutionary new treatment, according to doctors.

Alyssa, whose family would not give her last name, was diagnosed with acute lymphoblastic T-cell leukemia in 2021.

Conventional treatments, including chemotherapy and a bone marrow transplant, failed to prevent the disease from returning.

In May, Alyssa received universal CAR (chimeric antigen receptor) T cells that had been pre-made from a healthy donor, as part of a clinical trial.

Twenty-eight days later, she was in remission and was able to benefit from a second bone marrow transplant.

She is said to be “doing well” recovering at home, while her condition is being monitored by Great Ormond Street Hospital, where she received the treatment.

Alyssa’s mum Kiona said the family was “on strange cloud nine”, adding: “I hope this can prove that the research works and that they can offer it to more children – it all must have been for something.”

Alyssa, from Leicester, said: “Once I do it people will know what they need to do, one way or another, so it will help people – of course I will. TO DO.”

Without the treatment, the next step was palliative care

The pre-made cells were edited using new technology.

The modified CAR T cells can then be given to a patient to quickly find and destroy T cells in the body, including cancer cells.

Then the person may undergo a bone marrow transplant to restore their weakened immune system.

Without the treatment, Alyssa’s only next step would have been palliative care, the scientists said.

Dr Robert Chiesa, consultant in bone marrow transplantation and CAR T-cell therapy at GOSH, said the result was “pretty remarkable”, but said Alyssa’s condition should continue to be monitored over the next few years. coming months.

He said: “Since Alyssa fell ill with her leukemia in May last year, she has never achieved full remission – neither with chemotherapy nor after her first bone marrow transplant.

“It was only after receiving her CD7 CAR-T cell therapy and a second bone marrow transplant in GOSH that she became leukemia-free.”

“A finally better future for sick children”

Professor Waseem Qasim, Consultant Immunologist at GOSH, said: “This is a great demonstration of how, with expert teams and infrastructure, we can combine cutting edge technologies in the lab with real world results. hospital for patients.

“This is our most sophisticated cellular engineering to date and paves the way for other new treatments and ultimately a better future for sick children.”

The research will be presented at the annual meeting of the American Society of Haematology in New Orleans this weekend.

Great Ormond Street Hospital wants to recruit up to 10 T-cell leukemia patients who have exhausted all conventional options for a clinical trial. They will be referred by specialists.

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