The gene-editing therapy is expected to offer a cure for some people with the inherited blood disorder
A £1.65 million treatment has been approved for use for some NHS patients, offering hope of a cure to those with an inherited blood disorder. Campaigners have shared their joy as health officials approved a gene-editing therapy for certain children and adults with severe sickle cell disorder.
The only curative treatment currently available for people with sickle cell in the UK is a donor stem cell transplant. But it is not given to many patients due to the risks involved. Nice has endorsed the treatment for some people aged 12 and over who suffer from "recurrent vaso-occlusive crises" and have a specific genotype. Dr Samantha Roberts, Nice's chief executive, said: "Exa-cel could represent a potential cure for some people with severe sickle cell disease, freeing people from the burden of complications as well as addressing Nice’s aim of reducing health inequalities associated with the condition and getting the best care to patients fast.
"I had surgery on my spleen when I was six and a hip replacement at 22 – I will probably need another hip replacement in the next few months or years. “We are funding this new treatment option straight away so patients can benefit from the enhanced quality of life it offers.” “Previously, only individuals fortunate enough to have a stem cell donor match could access stem cell transplants as a potential cure.
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