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NICE recommends pioneering therapy for young leukaemia sufferers

Wednesday 21st November 2018
The National Institute for Health and Care Excellence (NICE) has backed a new leukaemia drug for use on patients aged under 25.
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The National Institute for Health and Care Excellence (NICE) has recommended a new treatment for young sufferers of relapsed or refractory B-cell acute lymphoblastic leukaemia (ALL) on the NHS via the Cancer Drugs Fund. 

It has backed the use of tisagenlecleucel, which is also known as Kymriah when made by Novartis, as a means of treating under-25s who have not responded to other treatments or who have suffered a relapse following a stem cell transplant.

The therapy works by taking the immune cells of a patient and modifying them with the drug in order to enable them to fight cancer cells. Having become CAR-T cells, they then attack and destroy the cancer. NICE said the treatment represents a potential cure for the condition. 

Around 25-30 people will be eligible for the treatment each year, with NHS England gearing up to make the therapy available within the next few weeks, with a specialist service being developed to manage access.  

Director of the Centre for Health Technology Evaluation at NICE Meindert Boysen, said: “NICE’s recommendation of tisagenlecleucel marks a new generation of personalised medicine that has the potential to transform the care of patients with cancer worldwide.

"CART-cell therapy is expensive and complex. We have worked in partnership with our stakeholders, NHS England and the company to make the therapy available to patients quickly."  

He added that a deal has been agreed with Novartis to lower the price of tisagenlecleucel so that patients in England can be "among the first in the world to access this exciting new treatment".

It would normally cost £282,000 for a single intravenous transfusion. NICE said the extent of the discount Novartis has agreed to is confidential. 

Director of research at the blood cancer charity Bloodwise Dr Alasdair Rankin said the decision by NICE will come as a "huge relief" for families fearing their children would be denied the treatment.
Written by James Puckle

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