The 13-year-old girl, identified only as Alyssa, was diagnosed with T-cell acute lymphoblastic leukemia in 2021. But her blood cancer did not respond to conventional treatment, including chemotherapy and a bone marrow transplant. She was enrolled on a clinical trial of a new treatment at London's Great Ormond Street Hospital for Children using genetically engineered immune cells from a healthy volunteer.
consultant, said her turnaround had been 'quite remarkable', although the results still needed to be monitored and confirmed in the next few months. Cutting edge Acute lymphoblastic leukemia is the most common kind of cancer in children and affects cells in the immune system, known as B cells and T cells, which fight and protect against viruses.
said Alyssa was the first patient known to have been given base-edited T cells, which involves chemically converting single nucleotide bases — letters of the DNA code — which carry instructions for a specific protein. Researchers at and University College London helped develop the use of genome-edited T cells to treat B-cell leukemia in 2015. But to treat some other types of leukemia the team had to overcome the challenge that T cells designed to recognize and attack cancerous cells had ended up killing each other during the manufacturing process. Multiple additional DNA changes were needed to the base-edited cells to allow them to target cancerous cells without damaging each other.
consultant immunologist and professor Waseem Qasim. 'It's our most sophisticated cell engineering so far and paves the way for other new treatments and ultimately better futures for sick children.' Alyssa said in the statement she was spurred to take part in the trial not just for herself but for other children. Her mother, Kiona, added: 'Hopefully this can prove the research works and they can offer it to more children.
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