Axicabtagene ciloleucel IV for relapsed/refractory acute lymphoblastic leukaemia in children and adolecents (aged 2-21 years) – second line

Acute Lymphoblastic Leukaemia (ALL) is a type of cancer affecting lymphocytes (a type of white blood cell), which results in overproduction of faulty lymphocytes. This means there are not enough healthy lymphocytes available to fight infection, increasing the risk of infections and bleeding. ALL most commonly occurs in children aged 2 to 5 years old. However it is considered a rare condition, affecting around 650 people per year in the UK. Some people will not respond to treatment (refractory) or some will respond to treatment but develop ALL again (relapsed). For those with relapsed or refractory ALL, there are limited treatments available.
Axicabtagene ciloleucel (KTE-C19) is new type of therapy where T cells (a type of immune cell) are altered to display receptors which recognise molecules on the surface of cancer cells and trigger the T cells to attack and kill the cancer cells. Axicabtagene ciloleucel is given to patients by a single infusion. If axicabtagene ciloleucel was licenced for use in children and adolescents with refractory/relapsed acute lymphoblastic leukaemia it would provide an additional, cancer specific treatment option for this population.