News and events

Great North Children's Hospital one of first in UK to provide pioneering cancer therapy for children


NHS England chief executive Simon Stevens has announced that children and young people in England will receive a groundbreaking cancer treatment, the first in what is expected to be a rapidly expanding class of personalised cancer therapies available on the NHS.

NHS England’s commercial deal with the manufacturer Novartis is the first in Europe, and comes less than 10 days after the treatment was granted its European marketing authorisation. It represents one of the fastest funding approvals in the 70 year history of the NHS.

The landmark deal means young people with a form of leukaemia are set to receive CAR-T (a chimeric antigen receptor T-cell) therapy within weeks, after NICE also green-lighted the treatment for entry into the reformed NHS Cancer Drugs Fund.

The Tisagenlecleucel form of CAR-T, also known as Kymriah, is the first in a wave of treatments in a new era of personalised medicine and part of the NHS’s long term plan to upgrade cancer services.

Unlike other forms of treatment, CAR-T therapy is specifically developed for each individual patient and involves reprogramming the patient’s own immune system cells which are then used to target their cancer. It has been shown in trials to cure some patients, even those with advanced cancers where other treatments have failed.

Dr Geoff Shenton, Consultant Paediatric Oncologist: “This is a huge announcement and it is really the start of a massive step forward in therapy for not just childhood leukaemia, but in future for other cancers.

“The majority of children with this type of leukaemia are cured with standard chemotherapy. This type of therapy is for those with a much more difficult to treat disease and for those that are not responding to treatment or where it comes back despite therapy.”

This will provide individualised treatment for cancer patients, by providing them with genetically modified immune cells. 

As Dr Shenton explains: “All of our cells in our body have various marks on their surface, these are sort of like flags that tell the body what that cell is and a part of what it does. What we do with this therapy is that the patient’s own immune cells are taken; they are modified to recognise the flags that are on the leukaemia cells. They are put back into the body where they would kill the leukaemia cells in the same way they would kill a cell infected with a virus.

“The technology is expanding rapidly and we are at a very early stage. We will see this therapy continue to develop, become more effective and diversify over time.”

Simon Stevens, chief executive of NHS England, initially made the announcement during a visit to the children's ward at the Great North Children's Hospital, RVI, as Newcastle upon Tyne Hospitals NHS Foundation Trust is one of the first three in the country to go through the international accreditation process for the provision of CAR-T therapy for children.

Subject to passing accreditation requirements the first treatments could begin in a matter of weeks.

“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. 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," he said.

Dr Alasdair Rankin, Director of Research at the blood cancer charity Bloodwise, said: “CAR-T cell therapy is the most exciting advances 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.”

Professor 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.”

Tisagenlecleucel, which costs around £282,000 per patient at its full list price, is licenced to treat patients up to 25 years old with B cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse. Patients in trials had an average age of 11.

The process of producing such a treatment is immensely complex and preparations are in their final stages. The first three NHS hospitals to go through the international accreditation process for the provision of CAR-T therapy for children are in London, Manchester and Newcastle. Subject to passing accreditation requirements the first treatments could begin in a matter of weeks.

Cancer survival in this country is now at an all-time high with 7,000 more people surviving cancer compared with three years ago, and the NHS is playing a leading role in the development and adoption of cutting edge technology in a new era of personalised medicine. So far, CAR-T has only been available to patients in Europe through clinical trials.

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