ESPE Yearbook of Paediatric Endocrinology

To read the full abstract: N Engl J Med 2017;377:1313-1315

“In 2010, 5-year-old Emily Whitehead was diagnosed with acute lymphoblastic leukemia.” So begins this remarkable story of how Emily became the first child to receive CART-19 (chimeric antigen receptor T-cell) therapy. She remains alive and well, and her successful treatment re-energised a whole line of research – the first CART therapy was licensed in US in August 2017. This perspective article describes the long and eventful research route towards successful cancer immunotherapy, which started in 1893 when surgeon William Coley found that streptococcus injection shrunk an inoperable osteosarcoma. As well as the wonderful insights into our powerful immune system, this article describes the methodical but also opportunistic nature of scientific progress, and the inventive but also determined characteristics of the pioneering scientists and families.