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A study published in the NEJM looks at universal base-edited CAR7 T cells for T-cell acute lymphoblastic leukemia.
Dr Tania Dexter, senior medical officer at UK stem cell charity Anthony Nolan, said:
“It’s encouraging to see the results of the phase 1 BE-CAR7 trial now released, three years since the first patient, Alyssa, was successfully treated with this unique cell therapy.
“The BE-CAR7 treatment is a world-first as it uses cutting edge ‘base editing’ technology to precisely modify several genes within T cells, helping them target T-cell acute lymphoblastic leukaemia (T-ALL). T-cell cancers have been challenging to target with typical CAR-T therapies, but this new treatment potentially addresses this challenge by adapting CAR T-cells to resist being destroyed by other CAR T-cells, while destroying cancerous T cells.
“The treatment is also unique in that it uses base editing to ensure donor T cells do not need to be precisely genetically matched to patients, allowing these manufactured cells to be ‘universal.’
“Donors from the Anthony Nolan stem cell register provided the cells to create the BE-CAR7 treatment. Our register of over 900,000 donors could help make more innovative therapies like this possible through research and cell therapy development.
“The results of the study are promising, with most patients achieving levels of remission allowing them to receive a stem cell transplant. Considering these patients had a low chance of survival before the trial, these results bring hope that treatments like this will continue to advance and become available to more patients.
“As with any novel cellular therapy, this phase 1 trial is just an initial indication of the effectiveness and safety of the treatment, and more work must be done to determine its wider clinical application. Yet the results are encouraging and demonstrate the recent leaps in technology that are allowing us to take on even greater challenges in the treatment of blood cancers and blood disorders.”
Prof Bob Phillips, Candlelighters Chair of Supportive Care Research for Children and Young People with Cancer, Professor of Paediatrics and Evidence Synthesis and Honorary Consultant in Paediatric Oncology, Hull-York Medical School, University of York, said:
“This is an important advance in the treatment of a highly-resistant subset of leukaemia. Where modified T-cell treatment (CAR-T) have been gaining some success in therapy for certain non-Hodgkin lymphomas and acute B-cell leukaemias, the technical challenge of creating T-cells which know how to attack the leukaemic T-cells, without attacking themself, has been a huge barrier. This case series from the London team shows how newly created CAR-T cells and a subsequent bone marrow transplant, have produced durable remissions, but are not a magic bullet for all relapsed T-cell disease.
“The therapy is not set to be a simple, one-shot, first line treatment for leukaemia. The long-term and late effects of having a transplant are varied and can be extremely challenging to live with, requiring life-long follow-up for the adverse effects of treatment (rather than the leukaemia itself). This is an important approach which allows us an extra line of therapy for some patients in highly specific situations.”
‘Universal Base-Edited CAR7 T Cells for T-Cell Acute Lymphoblastic Leukemia’ by Robert Chiesa et al. was published in NEJM at 21:30 UK time on Monday 8 December 2025.
DOI: 10.1056/NEJMoa2505478
Declared interests
Dr Tania Dexter: “Anthony Nolan donors provided the cells used to produce the BE-CAR7 treatment. We weren’t involved in the research in any other way.
Prof Bob Phillips: “None – don’t work with anyone in the group or have any financial or emotional connections with them or the labs involved.”