In 2010, 5-year-old Emily Whitehead from Philipsburg, Pennsylvania, USA was diagnosed with acute lymphoblastic leukaemia. This cancer affects B lymphocytes in the blood. After a year of increasingly intense chemotherapy, it was clear that Emily’s aggressive leukaemia was resistant to all available medicines. She was given only months to live. However, the Children’s Hospital of Pennsylvania was developing a new experimental immunotherapy for the treatment of acute lymphoblastic leukaemia and, in the spring of 2012, with all other options exhausted, 7-year-old Emily became the first child to receive this treatment.
The treatment was a living medicine that contained genetically modified versions of Emily’s own cells. Lymphocytes – T cells – had been taken from her blood and genetically modified in the lab. This enabled the T cells to make an artificial protein – a chimeric antigen receptor (CAR). These CAR T cells were grown in the laboratory to produce the billions of cells needed for Emily’s treatment. Eventually, after 2 weeks of work in the lab, the CAR T cells were injected into Emily’s bloodstream.
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