Teen ‘leukemia-free’ after revolutionary cell-editing therapy | Science and technology news

[ad_1]

Doctors say a 13-year-old boy became leukemia-free with the help of a revolutionary new treatment.

Alyssa, whose family did not want her surname to be revealed, was diagnosed with T-cell acute lymphoblastic leukemia in 2021.

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

In May, Alisa was given CAR (chimeric antigen receptor) pan-T cells previously made from a healthy donor, as part of a clinical trial.

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

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

Alyssa’s mother, Keona, said the family was “on weird cloud nine,” adding: “Hopefully this proves the research works and they can introduce it to more kids — it all has to be for something.”

“Once I do that, people will know what they need in one way or another, so doing that will help people – of course I will,” said Alyssa from Leicester.

Without treatment, the next step was palliative care

Previously manufactured cells were edited using a novel technique.

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

Then the person can have a bone marrow transplant to restore the depleted immune system.

Without treatment, the scientists said, the only next step for Alyssa was palliative care.

Dr Robert Kizza, a consultant bone marrow transplant and CAR T cell therapy at GOSH, said the outcome was “absolutely remarkable”, but said Alyssa’s condition should be monitored over the next few months.

He said: “Since Alyssa fell ill with leukemia in May last year, there has been no complete remission – not with chemotherapy and not after her first bone marrow transplant.

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

A better future for sick children

Professor Waseem Qasim, consultant immunologist at GOSH, said: “This is fantastic evidence of how cutting-edge technologies in the laboratory can be linked to real outcomes in hospital for patients, with expert teams and infrastructure.

“It is our most complex cell engineering yet and paves the way for other new therapies and ultimately a better future for sick children.”

The research will be presented at the American Society of Hematology annual meeting 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 clinical trials. They will be referred by specialists.

[ad_2]