A cancer immunotherapy will be offered on the NHS in England to some children and young adults with leukaemia, NHS England announced today.
Tisagenlecleucel (Kymriah) is one of a new line of immune-boosting treatments called CAR T cell therapies, which involve taking a patient’s own immune cells and modifying them in the lab. When the engineered cells are given back to the patient they are primed to recognise and attack the cancer cells.
The treatment will be given to patients up to the age of 25 with acute lymphoblastic leukaemia (ALL) whose cancer has either not responded or come back after standard treatments, including stem cell transplants.
“The majority of children with this form of blood cancer are cured with standard treatment,” said Professor Karl Peggs, a Cancer Research UK-funded immunotherapy expert. “But there remains a minority who do not respond to these therapies and have limited options available. Today’s announcement is great news for these children and young adults.”
“In the UK there will probably only be around 20 patients a year who might benefit from this CAR T cell therapy, which is a reflection of how well conventional treatments like chemotherapy work.”
In a clinical trial involving 75 people, 8 in 10 patients saw their disease completely disappear three months after treatment with the CAR T therapy and 76% of patients were alive a year later. But the treatment also led to serious side effects in 7 in 10 patients.
What happens now?
Kymriah will be made available via the Cancer Drugs Fund, which was set up to speed up access to promising new cancer treatments. This also gives the NHS and the drug company time to collect more data on the long-term benefits of the drug.
The treatment usually costs around £282,000 pounds per patient, but NHS England has reached a discount deal with the drug’s manufacturer, Novartis, which enables the treatment to be offered on the NHS. The deal between NHS England and Novartis is the first in Europe.
Professor Charles Swanton, Cancer Research UK’s chief clinician, said it was “fantastic news” for the small number of children and young people with this form of leukaemia who may benefit. These patients will be among the first in Europe to have routine access to CAR T cell therapy.
Kymriah is a complex treatment to give to patients because it uses a patient’s own immune cells, and hospitals need a have a special accreditation to offer it. NHS England announced today that it is preparing for the treatment to be available at specialist hospitals in London, Manchester and Newcastle. If all goes to plan, the first patient could be given the treatment in late autumn.
“It’s a very complicated treatment that can result in significant toxicity and because of this it will only be offered in a few specialist centres,” said Peggs.
A ‘new wave’ of personalised cancer therapies
The decision to approve the treatment comes a week after Kymriah and another CAR T cell therapy (Yescarta) were licensed for use in Europe.
Kymriah has also been tested in adults with an aggressive type of non-Hodgkin lymphoma for whom standard treatment has failed. The National Institute for Health and Care Excellent (NICE) is reviewing the evidence and will publish their draft recommendation later this year.
Last week Yescarta was provisionally rejected for use on the NHS in England. The NICE committee said that although clinical trial results suggest most patients respond to the treatment, there was not enough data comparing it with standard chemotherapy treatment. And it was too expensive to be considered cost-effective on the NHS.
The decision will be reviewed at the end of September.