Scientists at University College London (UCL) and Great Ormond Street Hospital (GOSH) have reported promising results from a world-first clinical trial using base-edited CAR T-cell therapy (BE-CAR7) to treat T-cell acute lymphoblastic leukemia (T-ALL), a rare and aggressive blood cancer. The therapy, which uses genome-edited immune cells from healthy donors, achieved deep remission in most of the ten patients treated—eight children and two adults—who had previously exhausted standard treatment options. The BE-CAR7 therapy employs base editing, an advanced form of CRISPR technology that alters single DNA letters without cutting the genome, reducing chromosomal risks. The modified T-cells target and destroy leukemic cells before patients undergo bone marrow transplants to rebuild their immune systems. The study, published in the New England Journal of Medicine and presented at the American Society of Hematology meeting, was led by Professor Waseem Qasim and supported by the UK’s Medical Research Council and NIHR. Researchers say the results mark a major step toward “off-the-shelf” gene therapies for resistant cancers, though they caution that outcomes vary and further trials are needed to confirm long-term safety and efficacy.
Sign in with Forget Password? Learn more December 8, 2025 by University College London edited by Sadie Harley, reviewed by Robert Egan scientific editor associate editor This article has been reviewed according t
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