First-of-its-kind 'designer' immune cells appear to clear baby girl's 'incurable' leukemia

  • 8 years ago
LONDON — A baby girl in Britain suffering from leukemia has become the first person in the world to receive an experimental gene editing procedure that miraculously reversed her cancer.

According to CBS News, Layla Richards had been diagnosed with Acute Lymphoblastic Leukemia (ALL) when she was just 14-week-old. Layla's family was losing hope after several failed treatments, until a team of doctors at London's Great Ormond Street Hospital performed an experimental gene editing procedure on her that saved her life.

This technology is known as TALEN, which had only been previously tested on mice. It uses molecular tools that act like scissors to cut specific genes in order to make the T-cells from healthy donors to behave in two specific ways: first, the cells are able to become invisible to a powerful leukemia drug that would normally kill them; second, they are reprogrammed to target and fight against leukemia cells only.

Layla spent several months in isolation due to her extremely weak immune system after the procedure. After the leukemia cells were confirmed to have been eliminated from Layla's body, she was given a bone marrow transplant to replace her entire blood and immune system, CBS News reported.

The treatment was prepared by scientists at London's Great Ormond Street Hospital, University College London and French biotech company Cellectis. Cellectis is going to fund full clinical trials of the therapy starting next year, Reuters reported.

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