NICE has recommended pioneering cancer treatment, CART-cell therapy, for people under the age of 25 with leukaemia, in final draft guidance.
Young people with relapsed or refractory B-cell acute lymphoblastic leukaemia (ALL) will now have access to tisagenlecleucel, also known as Kymriah made by Novartis, through the Cancer Drugs Fund (CDF).
The therapy, which involves taking a person’s own immune cells and modifying them to fight their cancer cells, has the potential to be a cure.
Tisagenlecleucel will be offered to people under the age of 25 who have not responded to current treatment or who have relapsed after stem cell transplant. Around 25 to 30 people will be eligible for tisagenlecleucel each year in England and a specialised NHS service is being developed to manage access to the new 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 said its is preparing to make tisagenlecleucel available in the coming weeks.
In September, it reached a commercial agreement with Kymriah’s manufacturer Novartis, paving the way for the NICE CDF recommendation.
The therapy, given as a single intravenous infusion, has a list price of £282,000, but the company have agreed a confidential discount.
NICE is currently reviewing two other CART-cell therapies from Kite, a Gilead company and Novartis for the treatment of diffuse large B cell lymphoma in adults.
Dr Alasdair Rankin, Director of Research at the blood cancer charity 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.”