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Innovative CAR-T therapy recommended to treat an aggressive form of leukaemia

Children and young adults with a form of leukaemia that develops rapidly are set to benefit after NICE today (Thursday, 11 April) recommended an innovative, life-extending treatment where the person’s own immune cells are modified to fight the cancer.

Tisagenlecleucel (also known as Kymriah and made by Norvartis) is recommended in final draft guidance for children and young adults up to and including those aged 25 with B-cell acute lymphoblastic leukaemia (ALL) which has not responded to treatment, relapsed after a stem-cell transplant, or relapsed for a second or later time. The treatment has been available through the NHS’s Cancer Drugs Fund since December 2018, with this new guidance now recommending its routine rollout.

ALL is a fast-developing type of blood cancer that affects some types of white blood cells, that are a vital part of people’s immune system. It causes the overproduction and accumulation of cancerous, immature white blood cells, also called lymphoblasts, to build up in a person’s bone marrow. This eventually means the body does not have enough white blood cells, red blood cells, or platelets to work properly.

Children and young people with ALL experience a range of debilitating symptoms including increased risk of infections, weight loss, fatigue, bruising and bleeding. If the disease progresses it can be fatal. It also significantly affects the ability of both the person and their caregivers to do daily tasks and maintain employment or education.

Tisagenlecleucel, is a CAR-T – chimeric antigen receptor T-cell – therapy where a patient’s own immune cells are taken and modified so they attach to and kill cancer cells. It is administered as a one-off infusion into the bloodstream.

It is estimated that around 40 children and young adults in England would be eligible for this innovative treatment. Around 300 people in England aged 25 and under are diagnosed each year with acute lymphoblastic leukaemia.

Tisagenlecleucel has been available within the NHS’s Cancer Drugs Fund (CDF) since December 2018. This meant people could have the treatment on the NHS while more data to resolve uncertainty about its effectiveness was collected ahead of this new evaluation by NICE.

The data collected on its use in the NHS and additional clinical trial evidence showed that people lived for longer and without experiencing the disease coming back or progressing, and improved overall survival for people compared with standard treatment.

Data gathered looking at tisagenlecleucel’s use in the NHS found that the 24-month overall survival was 72% following treatment.

Clinical trial evidence for tisagenlecleucel found the median overall survival was 48 months, compared with a median overall survival of 7.5 months for blinatumomab and a median overall survival of three months for salvage chemotherapy, two other standard treatments.

Confirmation of tisagenlecleucel’s effectiveness means NICE’s independent committee was able to recommend the treatment now be provided routinely within the NHS and it can be removed from the CDF.

Helen Knight, director of medicines evaluation at NICE, said: “I am delighted that we have been able to recommend continued use of this innovative treatment, which is already making a huge difference to the lives of children and young adults.

“The evidence from its use in the CDF and clinical trials shows it can offer an effective treatment, helping people live longer and with a better quality of life and could represent a potential cure for some people.

“Today’s news illustrates how NICE is determined to get the best care to patients fast, while ensuring value for money for the taxpayer.”

NHS England’s Cancer Drug’s Fund Lead, Professor Peter Clark, said: “It’s great news that this potentially curative drug is now being rolled out for routine use in the NHS for treating children and young people with an aggressive type of leukaemia.

“Tisagenlecleucel is just the latest drug to be recommended for routine use on the NHS and follows its use in certain circumstances for patients as part of our Cancer Drugs Fund since 2018, where it has shown its effectiveness in helping more children and young people live longer, healthier lives.”

The list price for tisagenlecleucel is £282,000 per infusion. The company has a commercial arrangement (simple discount patient access scheme). This makes tisagenlecleucel available to the NHS with a discount.

You can read the full final draft guidance for tisagenlecleucel for treating relapsed or refractory B-cell acute lymphoblastic leukaemia in people aged up to 25 years on the NICE website.