The first children to receive a game-changing personalised therapy for cancer will start treatment at Great Ormond Street Hospital in London this week.

A second centre, Royal Manchester Children’s Hospital, is also ready to start treating children with a rare form of leukaemia while a third, Newcastle upon Tyne Hospitals NHS Foundation Trust, is expected to join the programme next month.

CAR-T is a highly complex new type of immunotherapy which involves collecting and using the patients’ own immune cells to target their cancer in a process which is completed over a number of weeks.

The start of this treatment marks the beginning of a new era of personalised medicine, and forms part of the upgrade in cancer services which will be set out shortly NHS’s long term plan.

In September, NHS England struck the first full access deal in Europe on tisagenlecleucel, which can potentially cure some children with B cell acute lymphoblastic leukaemia (ALL) where other treatments have failed, enabling NICE to recommend the treatment for entry into the reformed NHS Cancer Drugs Fund last week.

The landmark deal with Novartis came less than 10 days after the treatment was granted its European marketing licence and represents one of the fastest funding approvals in the 70 year history of the NHS.

Simon Stevens, chief executive of NHS England said: “CAR-T shows immense promise, and the NHS is now leading from the front by ensuring patients in England among the first in the world to benefit. As we develop our Long Term Plan for cancer services this is one of the first in what is expected to be a growing number of personalised treatments available for NHS patients.”

Steve Powis, national medical director at NHS England, said: “As with any ground breaking new therapy, CAR-T is not without risk, but it does offer a real advance in our treatment of lymphoma and leukaemia for those who otherwise would have limited options.”

Unlike other forms of treatment, CAR-T therapy is specifically developed for each individual patient and has been shown in trials to cure some patients, even those with advanced cancers where other treatments have failed.

Dr Peter Steer, chief executive of Great Ormond Street Hospital said:  “I am delighted GOSH will be providing this pioneering treatment. As a leader in the field for developing personalised medicines for children and young people, GOSH has a proven track record in delivering this type of advanced cancer treatment. We aim to help every child we see fulfil their potential; offering CAR-T therapy does exactly that.”

Sir Michael Deegan CBE, chief executive of  Manchester University NHS Foundation Trust (MFT), said: “This new personalised therapy will deliver huge benefits to cancer patients and we are very proud to be leading the way nationally at MFT by being able to offer this revolutionary CAR-T treatment for both children and adults at the Royal Manchester Children’s Hospital and Manchester Royal Infirmary. This is a significant advancement in the treatment of cancer, offering new hope to patients when other treatment options have failed.”

Geoff Shenton, consultant paediatric haematologist at The Newcastle upon Tyne Hospitals NHS Foundation Trust, said: “We’re delighted that this revolutionary treatment is being offered to the first patients in London from as early as next week and that Newcastle will be in the position to provide CAR-T cell therapy very shortly afterwards.

“This is a step change in treatment, not just for childhood leukaemia but potentially other cancers in the future, and offers a genuine chance of a long-term cure for children and young people who have exhausted other treatment options.

NHS England will also today announce the hospitals which have received the go ahead to treat adults with the CAR-T axicabtagene-ciloleucel once NICE publish their recommendations.

For adults with lymphoma, University College London Hospitals NHS Foundation Trust, Manchester University NHS Foundation Trust (Manchester Royal Infirmary), The Christie NHS Foundation Trust and University Hospitals Birmingham NHS Foundation Trust have already received accreditation from JACIE, with King’s College Hospital NHS Foundation Trust, University Hospitals Bristol NHS Foundation Trust and Newcastle upon Tyne Hospitals NHS Foundation Trust all expected to complete the technical requirements needed to become accredited before the end of the year and be ready to receive patients.

Tisagenlecleucel for children with leukaemia

The tisagenlecleucel form of CAR-T, also known as Kymriah costs around £282,000 per patient at its full list price, is licenced to treat patients up to 25 years old with B cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse. Patients in trials of this treatment had an average age of 11. NHS England expect to treat 15-30 patients a year.

Axicabtagene-ciloleucel for adults with lymphoma

NHS England has also struck a deal on the CAR-T therapy, axicabtagene-ciloleucel, also known as Yescarta, which offers new hope for adult patients whose large cell lymphoma has returned or has stopped responding to previous treatment.

The treatment would have cost nearly £300,000 per patient at its full list price, but Gilead Sciences commercial agreement with NHS England will enable NICE to recommend its entry into NHS England’s Cancer Drugs Fund. Up to 200 patients a year will receive the treatment after NHS England negotiated a confidential deal with the manufacturer.


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