A cutting-edge personalised treatment for a form of childhood leukaemia is to be made available on the NHS in the first deal of its kind in Europe.
NHS England has negotiated a contract with pharmaceutical manufacturer Novartis for a therapy that reprogrammes a patient’s immune system to attack cancer cells.
The treatment, known as CAR-T (chimeric antigen receptor T-cell) therapy, will be available to children and young people under the age of 25 who have B cell acute lymphoblastic leukaemia.
The therapy, which is specifically developed for each patient and is currently only available in Europe in clinical trials, may be available within weeks and could help around 20 children a year.
In CAR-T therapy, cells are removed from the patient’s blood and reprogrammed into genetically-coded cells that “hunt” cancer cells.
A gene for a receptor that binds to protein in the cancer cells, a chimeric antigen receptor (CAR), is added in the laboratory and the cell is then reintroduced to the patient.
The process is specific to each patient and is part of a new wave of personalised medicines that have great potential for curing disease – but come at significant cost.
The Novartis treatment, known as Kymriah, has a list price of £282,000 per patient and was only approved by the National Institute for Health and Care Excellence (NICE) last week.
NHS England has said a commercial deal with Novartis was agreed over the weekend.
In a sign of the cost pressures facing the NHS, a separate CAR-T treatment for non-Hodgkin lymphoma was rejected by NICE on the grounds of cost.
Funding for the Novartis leukaemia treatment will be covered by the Cancer Drugs Fund and will be offered at one of three hospitals, in either Newcastle, Manchester or London.
NHS England chief executive Simon Stevens hailed the deal as part of a campaign to increase the availability of new treatments, while limiting the cost to the health service.
“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,” he said.
“Today’s approval is proof-positive that, in our 70th year, the NHS is leading from the front on innovative new treatments.
“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.”
Dr Alasdair Rankin, director of research at the blood cancer charity Bloodwise, said: “CAR-T cell therapy is the most exciting advance in treatment for childhood leukaemia for decades.
“Intensive chemotherapy can now cure the vast majority of children, but a significant number still tragically die every year because they do not respond to treatment.
“CAR-T cell therapy offers the genuine chance of a long-term cure for children who otherwise would have no other hope.”