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The UK's National Health Service (NHS) needs to treat children and young people with a costly new expensive drug that could transform the way the disease is treated.
NHS England CEO Simon Stevens announced an agreement with pharmaceutical company Novartis, which manufactures the immunotherapy drug under the name Kymriah.
The catalog price of the drug is £ 282,000 ($ 364,400) per patient and the treatment costs for the NHS could double. In the United States, the total cost of therapy can be up to $ 1 million.
Stevens and others have said that this form of cancer treatment, known as CAR-T therapy, is the future. It works by genetically engineering the killer T cells of the patient's immune system to recognize and destroy cancer cells.
CAR-T treatment is considered a "game changer" and cancer patients by the NHS will be among the first in the world to benefit. However, only 15 to 20 children with acute lymphoblastic leukemia (ALL) should be eligible for the drug. Only those who have failed a series of previous treatments, including stem cell transplants, will get it.
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Kymriah has also been allowed to treat adults with more common blood cancer, diffuse large B-cell lymphoma (DLBCL), but the National Institute for Health and Care has not yet decided whether the NHS can afford it.
The bill would be much higher, as around 200 adults could be eligible. A similar adult medicine, Yescarta, manufactured by Gilead, was denied because of the cost, which is US $ 373,000.
When Stevens revealed his intention to make HTR treatment available to the NHS in April, he asked Novartis to reduce the price of Kymriah. Any discount offered by the manufacturer is a trade secret.
CAR-T treatment should be developed for each patient. It involves taking blood and brainstorming the T cells of the patient's immune system to recognize and fight the cancer before transfusing it into the body.
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Clinical trials have yielded dramatic results, with response rates of over 80% in patients with advanced blood cancer. But there were also deaths, when the immune system of the patients reacted excessively to the therapy.
Alasdair Rankin, director of research at Bloodwise, a blood cancer charity, is pleased that children and young adults are receiving treatment. This use of CAR-T therapy is "only the visible part of the iceberg," he says, and there are other cancers, from solid myeloma to tumors, that it might help .
It compares the arrival of RTC-T treatment with that of radiotherapy, which has transformed cancer treatment and significantly improved long-term outcomes.
The production process of such a treatment is extremely complex, but the preparations are in their final phase, according to the NHS England. The first children could be treated in a few weeks. Three NHS hospitals are going through the international accreditation process for providing CAR-T treatment to children – in London, Manchester and Newcastle.
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