Teenager ‘leukemia-free’ after revolutionary cell-editing treatment | Science and technology news


A 13-year-old has become leukemia-free with the help of a revolutionary new treatment, doctors say.

Alyssa, whose family declined to give her last name, was diagnosed with T-cell acute lymphoblastic leukemia in 2021.

Traditional treatments, including chemotherapy and a bone marrow transplant, have failed to prevent the disease from coming back.

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

28 days later she was in remission and was able to receive a second bone marrow transplant.

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

Alyssa’s mum Kiona said the family is “on an odd cloud nine,” adding, “Hopefully this can prove the research is working and they can offer it to more kids – all of this 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 do that – of course I will do it.”

Without the treatment, the next step was palliative care

The prefabricated cells were processed with new technology.

The processed 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 can have a bone marrow transplant to restore their depleted immune system.

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

dr Robert Chiesa, bone marrow transplant and CAR-T cell therapy consultant at GOSH, said the result was “quite remarkable” but said Alyssa’s condition would need to be monitored further over the next few months.

He said: “Since contracting her leukemia in May last year, Alyssa has never achieved complete remission – not with chemotherapy and not after her first bone marrow transplant.

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

“Ultimately a better future for sick children”

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

“It is our most advanced cell engineering to date and paves the way for other new treatments and ultimately a brighter future for sick children.”

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

Great Ormond Street Hospital is looking to enroll up to 10 T-cell leukemia patients who have exhausted all conventional clinical trial options. They are referred by specialists.

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