US EditionGene-Edited Therapy Achieves 82% Remission in Leukemia Trial
BE-CAR7 yielded 82% deep remission in a trial of 11 patients with T-cell leukemia, enabling stem-cell transplants and with 64% disease-free up to three years.
- On Monday, the New England Journal of Medicine published results showing BE-CAR7 from University College London and Great Ormond Street Hospital produced 82% deep remissions enabling stem-cell transplants in nine children and two adults with T-ALL.
- Facing failed standard care, the trial focused on patients with T-cell acute lymphoblastic leukaemia whose chemotherapy and prior bone-marrow transplants had not worked, addressing urgent treatment needs.
- Scientists made three base edits to donor T-cells to disable self-targeting, remove the CD7 marker and render cells resistant, creating universal, off-the-shelf CAR T-cells that enable four-week clearance benchmark for transplant.
- Great Ormond Street Hospital Charity will support treatment for 10 more patients; Alyssa Tapley, first patient, has been discharged to long-term follow-up and returned to school.
- Researchers caution that this phase 1 trial is preliminary; some cancers lost the CD7 marker, raising infection risk during immune recovery and concerns over long-term transplant effects.
65 Articles
65 Articles
Gene-edited CAR-T cells erase aggressive T-cell leukemia
A cutting-edge therapy using base-edited immune cells is offering a major breakthrough for patients with one of the toughest forms of blood cancer, T-cell acute lymphoblastic leukaemia. By precisely rewriting tiny sections of DNA, scientists at UCL and Great Ormond Street Hospital created universal CAR T-cells capable of targeting the cancer without harming themselves—a long-standing challenge in T-cell–based therapies. Early trial results show …
What's the new treatment that can reverse incurable blood cancer?
The breakthrough in curing leukaemia was achieved by scientists at University College London (UCL) and Great Ormond Street Hospital (GOSH). They did so by finding a new way to utilise gene-edited immune cells from a donor to treat T-cell acute lymphoblastic leukaemia (T-ALL), which is a rare, fast-growing blood cancer. Here’s how they did it
This therapy, which at another time would have been considered typical of science fiction, managed to reverse a type of cancer in aggressive and incurable blood in several patients, according to doctors.The treatment consists of accurately editing the DNA of white blood cells to transform them into a “live drug” that fights cancer.The first girl who received the treatment, and whose history we published in 2022, is still free of the disease and …
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