US regulators approved the first gene therapy against cancer on Wednesday - a treatment that uses a patient's own immune cells to fight leukaemia.
The treatment is made by Novartis and is called Kymriah (tisagenlecleucel). This type of immunotherapy, known as a CAR-T cell therapy, was known by CTL019 until now.
"This marks the first CAR-T cell therapy to be approved anywhere in the world," said Novartis CEO Joseph Jimenez.
"It uses a new approach that is wholly personalised by using a patient's own T-cells."
Kymriah was approved by the US Food and Drug Administration for children and young adult patients up to the age of 25 with a form of acute lymphoblastic leukaemia.
The FDA described the approval as "a historic action" and a "new approach to the treatment of cancer and other serious and life-threatening diseases".
The treatment is not a pill or a form of chemotherapy, which can weaken the body's natural defences. Instead, it harnesses a patient's own immune cells, called T-cells and white blood cells, and trains them to recognise and fight cancer.
The patient's immune cells are removed with a special blood filtration process, sent to a lab and genetically encoded to be able to hunt down cancer cells. These re-engineered T-cells are transfused back into the patient, where they can begin attacking leukaemia.
Studies have shown that 83% of patients responded to the treatment, achieving remission within three months, Novartis said.
An application with the European Medicines Agency is expected to be filed by the end of the year.
The price of Kymriah - which is delivered to a patient just once - is $475,000 (R6.2-million), said Novartis.
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