A girl in Britain was cured of cancer, which was considered incurable: a new therapy showed brilliant results in the fight against oncology
British scientists have used a revolutionary new type of gene therapy to treat a young patient with a relapse of T-cell leukemia. The use of this technique - a world first - has raised hopes that it will soon help to cope with other childhood cancers. TheGuardian.
Alyssa, from Leicester, unsuccessfully underwent chemotherapy and a bone marrow transplant to alleviate her leukemia. With no further treatments available, the outlook for the 13-year-old was bleak.
But after being injected with donor T cells modified with a new technology known as baseline editing, Alyssa recovers and has been in remission for six months.
“Honestly, we are in 7th heaven with happiness. It's amazing,” said her mother, Kiona.
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Now the team at London's Great Ormond Street Hospital (Ghosh), who treated Alyssa, is preparing to recruit 10 more T-cell leukemia patients, who have also exhausted all conventional treatments, for further trials. If they are successful, it is hoped that the edited cells could be given to patients suffering from other types of leukemia and other diseases.
“This is our most sophisticated cell engineering to date, and it paves the way for other new treatments and ultimately a better future for sick children,” said immunology professor Wasim Qasim, one of the project leaders. He will present the results of the study at the American Society of Hematology meeting in New Orleans this weekend.
T-cell leukemia is a cancer that affects a class of white blood cells known as T cells. They do not develop properly and grow too fast, preventing the growth of blood cells in the body. Standard treatments include bone marrow transplantation and chemotherapy. In Alyssa's case, they were unable to stop the progression of the disease, and palliative care was her only option.
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But recent advances in cell therapy have offered a new treatment for her condition. The T cells were taken from a healthy donor and modified to kill other T cells, including her leukemic cells. This was done through basic editing, which allows scientists to make a single change to the billions of DNA letters that make up the human genetic code.
Other technologies can achieve such minor changes, but they come with a lot of side effects. Fewer basic editing issues and allowed Ghosh's team to make a number of individual changes to the donated T cells. These complex changes were necessary to ensure that the rearranged T cells would only attack the leukemic T cells, rather than destroy each other with friendly fire. They also allowed the cells to work after chemotherapy and also prevented them from affecting normal cells.
After conventional treatment, Alyssa never achieved a complete remission. After base-edited cell therapy and a second bone marrow transplant to restore her immune system, she was free of leukemia for over six months.
“The doctors said the first six months are the most important,” Kiona said. “We kept thinking, 'If they can just take the disease away for once, my daughter will be fine.' And perhaps we will be right."
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The important point is that Alyssa's therapy was based on donor T-cells, which can be edited, so matching a donor is not a problem. “This is a ready-made universal cell therapy, and if replicated, it would be a huge step forward in these types of treatments,” said Dr. Louise Jones of the Medical Research Council, which funded the project.
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