Saturday, November 7, 2015

Girl undergoes first-ever ‘gene-editing’ treatment

Layla, shown here at 16 months,
is the first patient to receive new
CANCER DIGEST – Nov. 7, 2015 – A one-year-old girl in London, England with leukemia is now cancer free and doing well as a result of a new treatment that uses ‘molecular scissors’ to edit genes and create designer immune cells.

The girl, named Layla, had relapsed acute lymphoblastic leukemia (ALL) that had failed to respond to conventional therapies and had limited treatment options available.

The patient’s parents were keen to try the treatment. Mother, Lisa, says: "We didn’t want to accept palliative care and so we asked the doctors to try anything for our daughter, even if it hadn’t been tried before."

A team at Great Ormond Street Hospital and UCL Institute of Child Health’s along with investigators at University College London and biotech company Cellectis had been developing ‘off-the-shelf’ banks of donor T-cells. The T-cells had been modified using molecular tools that act like scissors to splice specific genes into the cells to make the T-cells invisible to a powerful leukemia drug that would usually kill them, and reprograms them to only recognize leukemic cells.

The modified T-cells from donors, known as UCART19 cells, were delivered via intravenous line in around 10 minutes. After the cells had been delivered, the patient spent several months in isolation to protect her from infections while her immune system was extremely weak. 

The UCART 19 cells had been due to be used for final stage testing ahead of clinical trials in humans, but, after hearing about this infant, the team received special permission to try the new treatment early.

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