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Agence France-Presse

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.
Can gene editing cure viruses and cancer? A Hong Kong expert separates fact from fiction
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.
Cancer revolution as ‘living drug’ wins support from experts, paving way for first US gene therapy
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.
Gene therapy helps late-stage leukaemia patients for whom other treatments failed
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.


US approves ‘breakthough’ gene therapy for leukaemia

A 2012 photo of Emily Whitehead being checked by oncologist Stephan Grupp at the Children’s Hospital of Philadelphia. Photo: AP

In this July 9, 2015, photo, provided by Novartis Pharmaceuticals Corp., human T cells belonging to cancer patients arrive at Novartis Pharmaceuticals Corp.'s Morris Plains, N.J., facility. The Food and Drug Administration (FDA) approved the first treatment that genetically engineers patients’ own blood cells into an army of leukemia-fighting assassins. Manufacturer Novartis will create those turbocharged cells in this facility and ship them back to hospitals to infuse into patients. (Brent Stirton/Novartis Pharmaceuticals Corp. via AP)

This undated image made available by Novartis on Wednesday, Aug. 31, 2017 shows an IV bag of their drug Kymriah. On Wednesday, the Food and Drug Administration approved this first treatment that genetically engineers patients' own blood cells to seek and destroy childhood leukemia. (Novartis via AP)

Emily Whitehead catches fireflies in her backyard in Pennsylvania. MUST CREDIT: Photo by Sean Simmers for The Washington Post

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