Girl, 13, ‘leukemia-free’ after world’s first use of cell-engineered therapy

A 13-year-old girl who relapsed with a particular type of blood cancer is now leukemia-free after using for the first time in the world what scientists have described as the most sophisticated cell engineering to date.

Alyssa, whose family did not want to reveal her last name, said she believed taking the new, experimental treatment for the disease would help others, adding ‘of course I will’.

The scientists said that without the treatment, which came after chemotherapy and an initial bone marrow transplant failed to eliminate the cancer, her only next step would have been palliative care.

Alyssa, pictured here with family dog ​​Holly, is recovering at home (Family/PA handout)

Speaking about the breakthrough new therapy, Alyssa said: ‘Once I do that, people will know what they need to do, one way or another, so doing it will help people – of course I will.’

The teenager, from Leicester, received modified T-cells in the first-ever use of a modified cell therapy based on Great Ormond Street Hospital for Children (GOSH).

These prefabricated cells, from a healthy volunteer donor, have been modified using new technology to allow them to hunt down and kill cancerous T cells without attacking each other.

T lymphocytes are a type of white blood cell that move around the body to find and destroy faulty cells.

Alyssa, diagnosed with T-cell acute lymphoblastic leukemia (T-ALL) in 2021, has received all conventional treatments including chemotherapy and a bone marrow transplant, but the disease has returned.

She then became the first patient enrolled in a new clinical trial, funded by the Medical Research Council, in which she was given universal Chimeric Antigen Receptor (CAR) T cells that had been prefabricated from a healthy volunteer donor in May of this year .

Researchers have described basic editing as the chemical conversion of DNA code letters that contain instructions for a specific protein.

The modified CAR T cells can be given to a patient to quickly find and destroy T cells in the body, including cancerous ones, after which the person can have a bone marrow transplant to restore their depleted immune system.

Twenty-eight days after receiving the treatment, Alyssa was in remission, the researchers said, and was able to have a second bone marrow transplant.

She is said to be “well at home” as she recovers and continues with follow-up monitoring at GOSH.

It is hoped that the research, which is due to be presented for the first time at the American Society of Hematology’s annual meeting in New Orleans this weekend, could lead to new treatments and “ultimately a better future for sick children.” “.

Scientists aim to recruit up to 10 T-cell leukemia patients and have exhausted all conventional options for clinical trials in the new treatment.

GOSH doctors hope that, if successful, it could be offered to children in the early stages of treatment when they are less ill and could be used for other types of leukemia in the future.

Potential patients for trials will be referred by NHS specialists.

Professor Waseem Qasim, Consultant Immunologist at GOSH, said: “This is a great demonstration of how, with expert teams and infrastructure, we can connect cutting-edge technologies in the lab with real outcomes in the hospital for patients. It is our most sophisticated cell engineering yet and paves the way for other new treatments and ultimately a better future for sick children.

“We have a unique and special environment here at GOSH that allows us to rapidly scale up new technologies, and we look forward to continuing our research and bringing it to the patients who need it most.”

Alyssa’s mother, Kiona, said the family was “over the moon” and that it was “great to be home.”

She added: ‘I hope this can show that the research works and that they can offer it to more children – this must have all gone to something.’

Dr Robert Chiesa, bone marrow transplant and CAR-T cell therapy consultant at GOSH, said: “Since Alyssa fell ill with leukemia in May last year, she has never achieved a full with chemotherapy or after your first bone marrow transplant. transplant. Only after receiving her CAR-T CD7 cell therapy and a second bone marrow transplant in GOSH did she become leukemia free.

He described the result as “quite remarkable” but warned it needs to be monitored and confirmed in the coming months.

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