Approval for the first CAR-T treatment for ALL is renewed

Webpage last updated on: 11th March 2024

Treatment news

Today, tisagenlecleucel (Kymriah©) has been approved as a new treatment option on the NHS for patients under the age of 25 with acute lymphoblastic leukaemia (ALL) in England. It has been specifically approved for:  

• Patients who need further treatment due to relapse after a stem cell transplant OR 
• Patients who have relapsed twice or more from other treatments OR 
• Patient in whom existing treatment has not worked (this is known as refractory). 

It is also only approved for patients with the B cell form of ALL.  

This is excellent news, as tisagenlecleucel was first temporarily approved in 2018, funded by the Cancer Drugs Fund. Today’s decision confirms that this is now a treatment that will stay available on the NHS and be routinely available for those who meet the criteria.  

ALL in children is often highly curable, with survival rates of up to 90% at 5 years after diagnosis. However, for those who do relapse, further treatments options are needed. Current treatment options are not effective for all these patients, and tisagenlecleucel can now continue to be one of those options.   

Today’s news follows campaigning by several charities, including Leukaemia Care and Anthony Nolan. We emphasised the importance of new treatment options for these patients, using data from our previous surveys and case studies from patients.  

Our Policy and Evidence Manager, Charlotte Crowley, said: 

“Today’s decision will ensure ALL patients will continue to access this life saving treatment. These patients have few other options for treatment and CAR-T therapies have revolutionised the treatment of these patients already. We are really pleased that NICE has been able to approve this treatment for the longer term, giving patients and doctors much greater clarity on what is available if they do find themselves in this awful position. The severity of ALL which has not responded to treatment or that has relapsed after treatment was recognised when NICE gave the treatment its highest possible severity modifier. This means the NHS is willing to pay than they usually would for each year of health people gain when they access this treatment.” 

Additionally, our Advocacy Officer, Sophie Wheldon, attended the meeting where the decision was made. Prior to working at Leukaemia Care, Sophie was diagnosed with ALL when she was 20 and received CAR-T therapy when she was 21. Five years on from this, Sophie used her personal experience of the treatment, alongside her knowledge gained from working with other patients, to persuade the decision making committee of the value of this treatment for patients. 

Sophie said of today’s decision, “I’m very pleased that other ALL patients who receive the devastating news that they have relapsed will have access to this treatment. It saved my life and allowed me to start supporting other people who are going through such a life changing experience. I had already tried a stem cell transplant and various chemotherapies, which were sadly unsuccessful for me. Without receiving CAR T therapy, I simply would not be here today. This treatment represents a ray of hope for others in the same position that I was and I couldn’t be happier with the decision”.

To find out more about Sophie’s experience, click here.

The main clinical trial that was used to make a decision was the ELIANA trial. Data from this trial was already available when this treatment was initially assessed in 2018. But the results were too early to tell how long people would survive long term after treatment. Further data were collected since, both from the people who were on the ELIANA trial and those who had the treatment on the NHS whilst it was temporarily available. The data from the NHS showed that the treatment did work as effectively as what original trial data suggested it would, helping the committee to decide to approve tisagenlecleucel long term.  

How does this affect access to treatments elsewhere in the UK?

Decisions by the National Institute of Health and Care Excellence (NICE), such as today’s decision, only apply to England and Wales. This treatment will continue to be available here, thanks to today’s decision. However, CAR-T therapies are complex treatments requiring specialised staff and equipment. They are only delivered in certain locations at present and may require travel for you to receive them. Patients are also assessed by a panel of doctors for suitability for treatment, rather than by individual doctors.  

Northern Ireland usually chooses to adopt NICE recommendations. However, there are currently no CAR-T treatment centres in Northern Ireland. If you need CAR-T and you are suitable, you may be asked to travel for treatment. 

Scotland has a separate body that reviews treatments for use in NHS Scotland, the Scottish Medicines Consortium (SMC). This treatment was approved for NHS use in Scotland in 2019 and remains available in certain hospitals there.    

How does tisagenlecleucel work? 

Tisagenlecleucel is a chimeric antigen receptor T cell (CAR-T) therapy. This is a group of treatments that work by using a patients own blood cells, specifically their T cells. The cells are taken out the body, edited to make them better at fighting off cancer cells and then reinfused back into the patient.

You can read more about how CAR-T therapies generally work by clicking here.

How can I find out if  tisagenlecleucel is an option for me/someone I know? 

Your haematologist or clinical nurse specialist should explain to you when treatment will be needed and why they are recommending any treatment, including why other treatments are not suitable or available and why. They should also be clear that treatment is a choice for you to make and the expected consequences of your choices for your health.   

If you are struggling to know what to ask or how to get the information you need, we are also here to help. We can explain what treatments are available for people with your leukaemia, MPN or MDS, how treatment decisions are made or support you with speaking to your healthcare team.  

Whatever your worries, you can contact us via our helpline 08088 010 444,  or send your question to WhatsApp on 07500 068 065 . Alternatively you can email our team at support@leukaemiacare.org.uk (services available Monday to Friday 9am – 5pm).