Pioneering gene therapy approved for leukaemia in the US

PioneeringGeneTherapyApprovedForLeukaemiaInTheUS
Patient’s reprogrammed cells used as new therapy

Original source: New Scientist

A gene therapy for leukaemia has been given the green light by the US Food and Drug Administration (FDA). Announced last week, the approval is the first anywhere for CAR-T therapy – a type of reprogramming of immune cells that makes them better at targeting cancerous ones.

What the FDA approved was Kymriah, a treatment for B-cell acute lymphoblastic leukaemia, the most common childhood cancer in the US.

Made at a New Jersey facility operated by the pharmaceutical firm Novartis, Kymriah contains T-cells taken from the intended recipient. These immune cells will, however, have an added gene that codes for a protein called a chimeric antigen receptor (CAR), one that helps them home in on leukaemia cells.

In a trial, infusions of Kymriah achieved an 83 per cent remission rate over a period of three months in people who hadn’t responded to other treatments.

Kymriah is expected to cost $475,000, but could extend life by years rather than months.