'Ready-made' T-cell gene therapy tackles 'incurable' T-Cell leukaemia
2 days ago
- #gene therapy
- #T-cell leukaemia
- #medical breakthrough
- Groundbreaking gene-edited immune cell therapy developed by GOSH and UCL shows promise in treating T-cell acute lymphoblastic leukaemia (T-ALL).
- 82% of patients achieved deep remissions, with 63% remaining disease-free, some for up to three years.
- BE-CAR7 therapy uses base editing to modify donor T-cells, making them effective against cancerous T-cells without cutting DNA.
- Alyssa, the first patient treated, is now thriving and has been discharged to long-term follow-up.
- The therapy was developed with funding from NIHR, Wellcome, MRC, and GOSH Charity, and supported by Anthony Nolan donors.
- The research aims to provide better treatment options for the 20% of T-ALL patients who do not respond to standard therapies.
- GOSH Charity is funding an extended trial for 10 more patients as part of efforts to build a new Children's Cancer Centre.