From England's Great Ormond Street Hospital:
World first use of gene-edited immune cells to treat ‘incurable’ leukaemia
A new treatment that uses ‘molecular scissors’ to edit genes and create designer immune cells programmed to hunt out and kill drug resistant leukaemia has been used at Great Ormond Street Hospital (GOSH).
The treatment, previously only tested in the laboratory, was used in one-year-old, Layla, who had relapsed acute lymphoblastic leukaemia (ALL). She is now cancer free and doing well.
This breakthrough comes from GOSH and UCL Institute of Child Health’s (ICH) pioneering research teams, who together are developing treatments and cures for some of the rarest childhood diseases.
Chemotherapy successfully treats many patients with leukaemia but it can be ineffective in patients with particularly aggressive forms of the disease where cancer cells can remain hidden or resistant to drug therapy. Recent developments have led to treatments where immune cells, known as T-cells, are gathered from patients and programmed using gene therapy to recognise and kill cancerous cells. Multiple clinical trials are underway, but individuals with leukaemia, or those who have had several rounds of chemotherapy, often don’t have enough healthy T-cells to collect and modify meaning this type of treatment is not appropriate.
A bit more about the nuts and bolts of the treatment:
The treatment works by adding new genes to healthy donor T-cells, which arm them against leukaemia. Using molecular tools (TALEN®) that act like very accurate scissors, specific genes are then cut in order to make the T-cells behave in two specific ways. Firstly, the cells became invisible to a powerful leukaemia drug that would usually kill them and secondly they are reprogrammed to only target and fight against leukaemia cells.
The team at GOSH and the UCL ICH, along with investigators at University College London and biotech company Cellectis, had been developing ‘off-the-shelf’ banks of these donor T-cells and the first of which was due to be used for final stage testing ahead of clinical trials. But, after hearing about this infant, the team received special permission to try the new treatment early.
Very clever two-pronged attack - the off-the-shelf banks of cells is wonderful too. They can culture them as needed.
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