Overview
- Peer-reviewed results in the New England Journal of Medicine and a presentation at the ASH meeting report 82% very deep remissions and 64% disease-free status, with some patients off treatment for up to three years.
- The BE-CAR7 approach uses base editing to modify donor T cells by removing CD7 and CD52 and adding a CAR targeting CD7, creating universal cells that overcome fratricide and matching barriers.
- The therapy is designed as a bridge to curative transplant, with patients proceeding to stem cell transplantation once leukemia was eradicated within four weeks of infusion.
- The first recipient, Alyssa Tapley, treated in 2022, has been discharged to long-term follow-up, and a small cohort of children and adults at GOSH and King’s College Hospital achieved similarly strong responses.
- Reported side effects, including low blood counts, cytokine release syndrome and rashes, were manageable, though infection risk persisted until immune recovery, and GOSH Charity will support treatment for 10 additional patients as larger studies are pursued.