‘Game changing’ cancer immunotherapy treatment to be offered to UK child patients
US$1 million CAR-T therapy that engineers patient’s immune system T-cells to fight cancer is seen as the future of treatment for the disease. UK NHS patients will be among the first to have it
The UK’s National Health Service (NHS) is to treat children and young people with an expensive new cancer drug which may transform how the disease is treated.
NHS England chief executive Simon Stevens announced a deal with drug company Novartis, which makes the immunotherapy drug under the name Kymriah.
The list price of the drug is £282,000 (US$364,400) per patient and the therapy treatment costs for the NHS could double that. In the United States, the total cost of the therapy can reach US$1 million.
Stevens and others have said this form of cancer treatment, known as CAR-T therapy, is the future. It works by genetically engineering the patient’s own immune system’s killer T-cells to recognise and destroy cancer cells.
CAR-T therapy is seen as a “game changer” and NHS cancer patients will be among the first in the world to benefit. However, only 15 to 20 children with acute lymphoblastic leukaemia (ALL) are expected to be eligible for the drug. Only those who have failed a series of earlier treatments, including stem cell transplants, will get it.
Kymriah has also been licensed to treat adults with a more common blood cancer, diffuse large B-cell lymphoma (DLBCL), but the National Institute for Health and Care Excellence has not yet decided whether the NHS can afford it.
The bill would be much higher, as about 200 adults could be eligible. A similar drug for adults, Yescarta made by Gilead, has been turned down because of the cost, which is US$373,000 in the US.
When Stevens revealed his intention to make CAR-T therapy available to the NHS in April, he appealed to Novartis to reduce the price of Kymriah. Any discount the manufacturer has offered is a commercial secret.
CAR-T therapy has to be developed for each patient. It involves taking blood and engineering the patient’s own immune system T-cells to recognise and fight the cancer before transfusing them back into the body.
There have been spectacular results in clinical trials, with response rates in blood cancer patients with advanced disease of more than 80 per cent. But there have also been deaths, when patients’ immune systems have overreacted to the therapy.
Alasdair Rankin, the director of research at the blood cancer charity Bloodwise, is pleased that children and young adults will get the treatment. This use of CAR-T therapy is “only the tip of the iceberg”, he says, and there are other cancers, from myeloma to solid tumours, that it could help.
He likens the arrival of CAR-T therapy to that of radiotherapy, which transformed cancer treatment and substantially improved long-term outcomes.
The process of producing such a treatment is immensely complex but preparations are in their final stages, according to NHS England. The first children could be treated within weeks. Three NHS hospitals are going through the international accreditation process for the provision of CAR-T therapy for children – in London, Manchester and Newcastle.