British scientists have used a revolutionary new type of gene therapy to treat a young patient with relapsed T-cell leukemia. The use of this technology is a world first, raising hopes that it could soon help fight other types of cancer and serious childhood illnesses.Alyssa from Leicester underwent chemotherapy and a bone marrow transplant to alleviate her leukemia, but to no avail. With no other treatment available, the 13-year-old girl’s prospects were bleak.But after Alyssa was given her IV of donated T cells that had been modified with a new technique called base-editing, she recovered and has been in remission for six months. “To be honest, we’re on weird Cloud Nine. It’s unbelievable,” said her mother, Kiona.
Now, the team at London’s Great Ormond Street Hospital (Gosh), which treated Alyssa, is another of her 10 T-cell leukemia patients who have exhausted all conventional treatments for further study. I am preparing to recruit. If successful, the base-edited cells could be administered to patients with other types of leukemia and other diseases.One of his project leaders, an immunologist Professor Waseem Qasim, said, “This is the most sophisticated cell technology to date, and will pave the way for other new therapeutics and, ultimately, It gives sick children a better future.” He plans to present his findings 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, grow rapidly, and affect the growth of blood cells in the body. Standard treatment includes bone marrow transplantation and chemotherapy. In Alyssa’s case, these did not stop the progression of the disease, and palliative care seemed to be her only option. But recent advances in cell therapy have provided new ways to treat her condition.
T cells were taken from healthy donors and designed to kill other T cells, including leukemic cells. rice field. This was achieved through basic editing that allowed scientists to make a single change to the billions of letters of DNA that make up the human genetic code.\Other technologies can achieve such small changes, but with more side effects. This was not a basic editing problem and the Gosh team was able to make numerous individual edits to her T cells provided. These complex modifications were necessary to ensure that retargeted T cells attacked only leukemia T cells and did not destroy each other through friendly fire. They kept cells functioning after chemotherapy and prevented normal cells from being affected.Importantly, Alyssa’s treatment was based on donated T cells. Her T cells can be edited, so donor matching is not an issue. “This is an off-the-shelf universal cell therapy that, if replicated, would be a major step forward in these types of treatments,” said Louise Jones of the Medical Research Council, who funded the project.
