US approves ‘breakthough’ gene therapy for leukaemia
The US approved the first gene therapy against cancer on Wednesday – a treatment that uses a patient’s own immune cells to fight leukaemia – opening a new era in the fight against one of the world’s top killers.
The treatment is made by Novartis and is called Kymriah (tisagenlecleucel).
This type of anti-cancer immunotherapy, known as a CAR-T cell therapy, was known by CTL019 until now.
“This marks the first ever CAR-T cell therapy to be approved anywhere in the world,” Novartis CEO Joseph Jimenez told reporters. “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 age 25 with a form of acute lymphoblastic leukaemia (ALL).
To qualify for treatment, patients must have B-cell precursor ALL that is refractory, or the patient has relapsed at least twice.
The FDA described the approval as “a historic action” that would usher “in a new approach to the treatment of cancer and other serious and life-threatening diseases,” said a statement.
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, 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 with a viral vector to be able to hunt down cancer cells.
These re-engineered T-cells are then transfused back into the patient, where they can begin attacking leukaemia.
Studies have shown that 83 per cent of patients responded to the treatment, achieving remission within three months, Novartis said.
An application with the European Medicines Association is expected to be filed by the end of the year.
The treatment was pioneered by Carl June at the University of Pennsylvania.
Its most high-profile patient is Emily Whitehead, now 12, who six years ago was the first child to receive what was widely considered a risky treatment.
She has been cancer free since.
In 2014, US regulators designated CTL019 as a “breakthrough therapy” and put the experimental immunotherapy agent on the fast track to market approval.
It was the first cancer immunotherapy to receive the breakthrough designation. More are expected to follow in the coming years as the field of immunotherapy grows.
Last month, an advisory panel to the FDA unanimously urged approval of the cutting edge cancer therapy.
About two dozen centres in the US are expected to be able to treat patients seeking Kymriah, with as many as 35 up and running by the end of the year.
About 600 patients in the US meet the criteria of being under 25 and having relapsed or refractory ALL, according to Novartis.