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Doing Well

Two children who received an experimental gene-editing therapy to treat their leukemia are doing well a year on, New Scientist reports.

Investigators from the Great Ormond Street Hospital in London and elsewhere treated an 11-month-old girl named Layla with CAR-T cell therapy in June 2015 and later that year, in December, they also treated a 16-month-old; both infants had refractory relapsed B cell acute lymphoblastic leukemia. As Paul Veys from Great Ormond and University College London and his colleagues report in Science Translational Medicine this week, both children reached molecular remission within 28 days.

In particular, the treatment team used a therapy developed by Cellectis in which transcription activator-like effector nucleases (TALENs) are used to edit T cells to express a chimeric antigen receptor against CD19, a leukemia antigen found on B cells. TALENs also disrupted the TCR and CD52 genes to make the treatment universal and able to be used in unmatched individuals.

While both patients did develop signs of graft-versus-host disease, New Scientist notes the team was on the look out for it and treated it successfully as it arose.

New Scientist notes that a number of labs are pursuing gene editing to improve cancer immune therapies, particularly with the rise of CRISPR-based editing.