World’s first “designer” gene therapy reversed lymphoblastic leukaemia of a one year old girl
Layla Richards is the first person in the world to receive a pioneering genetic therapy for the treatment of terminal lymphoblastic leukaemia. Layla was diagnosed with acute lymphoblastic leukaemia at 3 month old. Layla since then went through extensive chemotherapy treatment and bone marrow transplant, but the cancer proved persistent. She was advised to go through palliative care before undergoing this highly expermental therapy that had only been trialled in mice before.
The treatment, originally developed by a french biotech company, Cellectis, involves arming blood cells taken from a healthy donor with cancer-fighting genes. Genes in these donor immune cells were precisely modified by “microscopic scissors”, a technology known as Talens. The cells were artificially designed to add certain genes to seek out and kill leukaemia cells, meanwhile to switch off other genes to make them undetecable by drugs and immune cells in her system. Layla was given an infusion of 50 million of the genetically-engineered cells which cleared the majority of the cancer cells in 3 months. She was then given a bone marrow transplant to restore her immune system.
TALENs (transcription activator-like effector nucleases) is a milestone in the development of gene-editing technologies which precisely target and alter DNA sequences. This is the first time that cells edited using TALENs have been given to a person and only the second time in the world any gene-edited cells have been used.
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