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.
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64 Articles
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 …
Genome-edited immune cell therapy shows promise for treating aggressive blood cancer
A groundbreaking new treatment using genome-edited immune cells, developed by scientists at UCL (University College London) and Great Ormond Street Hospital (GOSH), has shown promising results in helping children and adults fight a rare and aggressive form of blood cancer called T-cell acute lymphoblastic leukemia (T-ALL).
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