28 November 2017 - It’s a diagnosis that no parents want to hear - their child has acute lymphoblastic leukaemia. 

While a treatable cancer of the bone marrow and blood, traditional drug treatment regimens can be brutal and not always successful. But, in August the FDA announced the approval of a new gene therapy to treat acute lymphoblastic leukaemia (ALL). Known as Kymriah (tisagenlecleucel), this Novartis product is the first gene therapy approved in the U.S., thereby opening a new era in medicine.

The value of Kymriah has been wonderfully captured by Dr. Lisa Rosenbaum in the New England Journal of Medicine, where she outlines the development of CAR-T technology – the science on which Kymriah is based. Dr. Rosenbaum, however, does more than talk about the underlying science. She puts this research into context with the story of Emily Whitehead who, at the age of five, was diagnosed with ALL. Emily was put through the rigors of multiple rounds of chemotherapy to no avail. Emily’s physicians told her parents to consider hospice, but instead, they entered her in a clinical trial for Novartis’ Kymriah. The procedure worked and Emily is now a thriving 12 year-old.

Read Forbes magazine article