Emily Whitehead
The first child treated with CAR-T cells, in 2012. Her recovery from relapsed leukaemia turned an experimental idea into an approved therapy.
Emily Whitehead was diagnosed with acute lymphoblastic leukaemia in 2010 at the age of five and relapsed twice on standard chemotherapy. In April 2012, at six, she became the first child in the world to receive CD19-directed CAR-T cells (CTL019, later tisagenlecleucel), developed at the University of Pennsylvania and given at Children's Hospital of Philadelphia. She developed severe cytokine release syndrome, which was controlled with the IL-6 blocker tocilizumab, a rescue that became standard practice for CAR-T toxicity. She has been in remission since May 2012. In August 2017 the FDA approved tisagenlecleucel, the first gene-modified cell therapy for any disease, for children and young adults with relapsed or refractory ALL. Her parents founded the Emily Whitehead Foundation to support paediatric cancer immunotherapy research.
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- Key paperMaude 2014: CD19 CAR-T cells produce complete remission in 27 of 30 children and adults with relapsed ALL
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