MIAMI • US regulators have approved the first gene therapy against cancer - a treatment using 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 Swiss drugmaker Novartis and is called Kymriah (tisagenlecleucel). This type of anti-cancer immunotherapy is also known as CAR-T cell therapy.
"This marks the first-ever CAR-T cell therapy to be approved anywhere in the world," said Novartis chief executive Joseph Jimenez.
"It uses a new approach that is wholly personalised by using a patient's own T-cells."
Kymriah was approved on Wednesday for patients up to 25 years of age for an aggressive type of leukaemia - B-cell acute lymphoblastic leukaemia - that has resisted standard treatment or relapsed.
The Food and Drug Administration (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 through 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 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.
The price of Kymriah - which is delivered to a patient just once - is US$475,000 (S$645,000), said Dr Bruno Strigini, oncology chief executive at Novartis. Patients who do not respond to the treatment within the first month would not be expected to pay, he said.
The more common treatment for leukaemia - bone marrow transplants - can cost between US$540,000 and US$800,000 in the first year in the United States, Dr Strigini said.
The treatment was pioneered by Dr Carl June at the University of Pennsylvania.
Its most high-profile patient is Emily Whitehead, now 12. Six years ago, she was the first child to receive what was widely considered a risky treatment. She has been cancer-free ever since.
However, Kymriah will carry a boxed warning because of the treatment's potential to cause deadly side effects - including neurological complications and what is known as cytokine release syndrome, a systemic reaction triggered by the destruction of the cancer cells.
AGENCE FRANCE-PRESSE, BLOOMBERG