Children with 'no other hope' to receive groundbreaking cancer treatment on NHS after funding deal

Therapy 'a true game changer', says health chief Simon Stevens

Tisagenlecleucel, a form of chimeric antigen receptor T-Cell (CAR-T) therapy, has been shown to cure some patients with B cell acute lymphoblastic leukaemia (Getty/iStock)

Children and young people with a form of leukaemia will have access to a groundbreaking treatment after NHS England secured a deal with the drug manufacturer.

Tisagenlecleucel, a form of chimeric antigen receptor T-Cell (CAR-T) therapy, has been shown to cure some patients with B cell acute lymphoblastic leukaemia.

NHS patients will be the first in Europe to have routine access to the treatment, which costs £282,000 per patient at full list price, following the agreement with pharmaceutical company Novartis.

The deal comes less than 10 days after the therapy was granted European marketing authorisation and represents one of the fastest funding approvals in the history of the health service, NHS England said.

The deal will be announced by Simon Stevens, chief executive of NHS England, at the Health Innovation Expo in Manchester on Wednesday.

“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,” Mr Stevens will say.

“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.”

CAR-T therapy is a personalised treatment, which reprogrammes a patient’s immune system cells to target the cancer.

Tisagenlecleucel, also known as Kymriah, has been shown in trials to “cure” some patients, even those with advanced cancers who have not responded to other treatments, NHS England said.

The therapy is licensed for use in patients up to the age of 25 with B cell acute lymphoblastic leukaemia that is refractory, in relapse post-transplant or in second or later relapse.

It has been approved for use by the National Institute for Care and Excellence through the NHS Cancer Drugs Fund.

Three UK hospitals in London, Manchester and Newcastle are awaiting approval to provide CAR-T therapy and, if successful, could begin treating patients with Tisagenlecleucel within weeks, NHS England said.

Dr Alasdair Rankin, director of research at 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.”

PA