A pioneering cancer treatment has been recommended for leukaemia patients under the age of 25.
CART-cell therapy involves taking a person’s own immune cells and modifying them to fight their cancer cells, and has the potential to be a cure.
The National Institute for Health and Care Excellence (Nice) said young people with relapsed or refractory B-cell acute lymphoblastic leukaemia (ALL) will have access to tisagenlecleucel through the Cancer Drugs Fund (CDF).
Blood cancer charity Bloodwise said the announcement will come as a “huge relief for a number of worried families” and give children with leukaemia the real possibility of long-term survival.
Tisagenlecleucel will be offered to people under the age of 25 who have not responded to current treatment or who have relapsed after a stem cell transplant.
Around 25 to 30 patients will be eligible for tisagenlecleucel each year in England and a specialised NHS service is being developed to manage access to the therapy.
Young people with relapsed or refractory B-cell ALL have repeated cycles of treatment, which can have substantial psychological and physical effects and some have poor outcomes. The current treatment for this stage of the disease is blinatumomab or chemotherapy.
NHS England is preparing to make tisagenlecleucel – also known as Kymriah and made by Novartis – available in the coming weeks.
Meindert Boysen, director of the Centre for Health Technology Evaluation at Nice, said: “Nice’s recommendation of tisagenlecleucel marks a new generation of personalised medicine that has the potential to transform the care of patients with cancer worldwide.
“CART-cell therapy is expensive and complex. We have worked in partnership with our stakeholders, NHS England and the company to make the therapy available to patients quickly.
“Novartis have agreed to offer tisagenlecleucel at a lower price so that people using the NHS can be among the first in the world to access this exciting new treatment.”
Alasdair Rankin, director of research at Bloodwise, said: “CART-cell therapy can give children with leukaemia the real possibility of long-term survival if they do not respond to standard treatments.
“Today’s announcement will come as a huge relief for a number of worried families. We hope that people will be able to access the therapy as soon as possible.”
The therapy, given as a single intravenous infusion, has a list price of £282,000, but Novartis has agreed a confidential discount.
NHS England’s director of specialised commissioning, John Stewart, said: “NHS cancer patients will now be amongst the first in the world to benefit from this game-changing therapy.
“Constructive engagement with Nice and fast-track negotiations with NHS England shows how responsible and flexible life science companies can succeed – in partnership with the NHS – to make revolutionary treatments available to patients.”
Nice is reviewing two other CART-cell therapies for the treatment of diffuse large B cell lymphoma in adults.