New Cell Therapy Clears Leukemia in Dying Baby

A baby whom doctors thought almost certain to die has been cleared of a previously incurable leukemia in the first human use of an “off-the-shelf” cell therapy from Cellectis that creates designer immune cells.

One-year-old Layla had run out of all other treatment options when doctors at Britain’s Great Ormond Street Hospital (GOSH) gave her the highly experimental, genetically edited cells in a tiny 1-milliliter intravenous infusion.

Two months later, she was cancer-free and she is now home from hospital, the doctors said at a briefing about her case in London on Wednesday.

“Her leukemia was so aggressive that such a response is almost a miracle,” said Paul Veys, a professor and director of bone marrow transplant at GOSH who led the team treating Layla.

“As this was the first time that the treatment had been used, we didn’t know if or when it would work, so we were over the moon when it did.”