A 13-year-old girl who relapsed with a particular type of blood cancer is now leukemia-free after a world-first use of what scientists have described as the most sophisticated cell engineering to date.
Alyssa, whose family declined to give her surname, said she believed taking the experimental new 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 clear her cancer, her only next step would have been palliative care.
Speaking about the revolutionary new therapy, Alyssa said: “Once I do this, people will know what they need to do, one way or another, so it will help people – of course, I will do it.”
The teenager, from Leicester, received basic modified T cells in the first ever use of basic modified cell therapy at Great Ormond Street Hospital for Children (GOSH).
These pre-made cells, sourced from a healthy volunteer donor, have been modified using new technology to allow them to track down and kill cancerous T cells without attacking each other.
T cells are a type of white blood cells that travel around the body to find and destroy defective cells.
Alyssa, who was diagnosed with T-cell acute lymphoblastic leukemia (T-ALL) in 2021, received all conventional treatments, including chemotherapy and a bone marrow transplant, but the disease recurred.
She then became the first patient enrolled in a new clinical trial, funded by the Medical Research Council, in which she received universal CAR (Chimeric Antigen Receptor) T-cells that had been pre-made from a healthy volunteer donor. health in May this year. .
The researchers described base editing as the chemical conversion of DNA code letters that carry instructions for a specific protein.
Modified CAR T cells can be given to a patient to quickly find and destroy T cells in the body, including cancer cells, after which the person can undergo a bone marrow transplant to restore their weakened immune system.
Twenty-eight days after receiving the treatment, Alyssa was in remission, the researchers said, and was able to undergo a second bone marrow transplant.
She is said to be “doing well at home” as she recovers and continues follow-up monitoring at GOSH.
It is hoped that the research, which is due to be presented for the first time at the annual meeting of the American Society of Hematology in New Orleans in the United States this weekend, could lead to new treatments and “a finally a better future for sick children”.
The scientists aim to enroll up to 10 patients with T-cell leukemia and have exhausted all conventional options for the clinical trial in the new treatment.
Doctors at GOSH hope that if successful, it can be offered to children earlier in their treatment when they are less ill and that it can be used for other types of leukemia in the future.
Potential patients for the 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 combine cutting-edge technologies in the laboratory 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.
“We have a unique and special environment here at GOSH that allows us to rapidly develop new technologies and we look forward to continuing our research and bringing it to the patients who need it most.”
Alyssa’s mum Kiona said the family was “on a weird cloud nine” and it was “amazing to be home”.
She added: “I hope this can prove that the research works and that they can offer it to more children – it must all have been for something.”
Dr Robert Chiesa, consultant in bone marrow transplantation and CAR T-cell therapy at GOSH, said: “Since Alyssa fell ill with her leukemia in May last year, she has never had a complete 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.
He called the result “quite remarkable”, but warned it needed to be monitored and confirmed over the coming months.