Baby With Incurable Leukemia Cured With Designer Immune Cells

By Kate Kelland

LONDON (Reuters) – 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.”

The gene-edited cell treatment was prepared by scientists at GOSH and University College London (UCL) together with the French biotech firm Cellectis, which is now funding full clinical trials of the therapy due to start next year.

It is designed to work by adding new genes to healthy donated immune cells known as T-cells, which arm them against leukemia.

Using a gene-editing technology called TALEN, which acts as “molecular scissors,” specific genes are then cut to make the T-cells behave in two specific ways: Firstly, they are rendered invisible to a powerful leukaemia drug that would usually kill them and secondly they are reprogrammed to only target and fight against leukemia cells.

Other drugmakers including Novartis, Juno Therapeutics and Kite Pharma have tested genetically modified T-cells extracted from an individual patient. However, this is the first time cells from a healthy donor have been used in a process —> Read More