NHS to treat young cancer patients with expensive 'game changer' drug

The NHS is to treat children and young people with an expensive new cancer drug which has the potential to transform how the disease is treated.

Simon Stevens, the NHS England chief executive, will announce on Wednesday that a deal has been done with the drug company Novartis, which makes the immunotherapy drug under the name Kymriah.

How does CAR-T therapy work?

CAR-T therapy is a new type of immunotherapy. The novel idea is to collect T-cells from the blood of the patient and engineer them to recognise the cancerous cells that have been hiding in the body unnoticed and that they have failed to destroy.

T-cells are lymphocytes or white blood cells. They are key players in the immune system, moving around the body to attack infection and diseases. 

They should attack a cancer, but sometimes fail to identify cancerous cells as the threat they are. CAR-T therapy aims to teach the T-cells to recognise and attack the target.

A child with acute lymphoblastic leukaemia (ALL) which has returned in spite of a number of different treatments would be eligible for CAR-T therapy (CAR stands for chimeric antigen receptor and the T is for T-cell).

 The first step is to insert a tube in each arm. Blood is then withdrawn from one arm and T-cells are removed by passing it through an apheresis machine. The rest of the blood returns to the body through the tube in the other arm. 

In a lab, the T-cells are genetically engineered to recognise and target a specific protein on the cancer cells. The CAR-T cells, as the changed cells are now called, multiply in the lab, while the patient is given chemotherapy to kill off any remaining T-cells in the body.

 Then the child is given a transfusion of CAR-T cells, which will hopefully attack and kill the cancer cells in the blood. 

The list price of the drug is £282,000 per patient and treatment costs for the NHS could double that. In the United States, the total cost of the therapy can reach $1m.

But 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 a true game changer and NHS cancer patients are now going to be amongst the first in the world to benefit,” Stevens will say in a speech at the Health and Care Innovation Expo in Manchester.

However, only 15 to 20 children with acute lymphoblastic leukaemia (ALL) are expected to be eligible for the drug. It will be given only to those who have failed a series of earlier treatments, including stem cell transplants.

Kymriah has also been licensed to treat adults with a more common blood cancer, diffuse large B-cell lymphoma (DLBCL), but a decision is yet to be made by the National Institute for Health and Care Excellence (Nice) on whether the NHS can afford it. The bill would be substantially higher because 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 $373,000 (£290,000) in the US.

When Stevens revealed his intention to make CAR-T available to the NHS in April, he appealed to Novartis to reduce the price of Kymriah. Any discount that 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 over 80%. 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, said he was very pleased that children and young adults would get the treatment. “It is very exciting for children with leukaemia,” he said.

 This use of CAR-T therapy was “only the tip of the iceberg”, he said, and there were other cancers, from myeloma to solid tumours, that it could help. He likened the arrival of CAR-T therapy to that of radiotherapy, which transformed cancer treatment and substantially improved long-term outcomes.

Prof Charles Swanton, Cancer Research UK’s chief clinician, said: “It’s fantastic news for children and young people with this form of leukaemia that CAR-T cell therapy will be made available on the NHS, making them the first in Europe to have routine access to this exciting new type of immunotherapy. We applaud NHS England, Nice and the company for working together to make this immensely complex treatment available to patients quickly, through the Cancer Drugs Fund.”

The process of producing such a treatment is immensely complex but preparations are in their final stages, according to NHS England, and 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.

“Today’s approval is proof-positive that, in our 70th year, the NHS is leading from the front on innovative new treatments,” Stevens will say. “This constructive fast-track negotiation also shows how responsible and flexible life sciences companies can succeed – in partnership with the NHS – to make revolutionary treatments available to patients.” - The Guardian , UK