A “groundbreaking” £1.65m treatment offering a potential cure for people in England living with sickle cell disease has been approved for use on the NHS, the medicines watchdog has announced.
Campaigners welcomed news of the approval of the one-time gene therapy, known as exagamglogene autotemcel, or exa-cel, which edits the faulty gene in a patient’s own stem cells.
The NHS estimates that 50 patients, older children and adults with a severe form of the disease, who are suitable for a stem cell transplant but without a matched donor, will each year receive the cutting-edge treatment.
Clinical trials for exa-cel have indicated that the therapy can stop painful sickle cell crises, which occur when blood vessels become blocked, with researchers finding that there was a “functional cure” in 96.6% of participants who received the treatment.
The National Institute for Health and Care Excellence originally rejected the treatment last March, saying it needed further detail regarding the effectiveness of the treatment.
The condition is more prevalent in people with an African or Caribbean family background, according to the NHS. In England, about 17,000 people are living with the disease. Of those, 4,000 are believed to be eligible for the new treatment.
Sickle cell disease changes the shape of blood cells into crescents, hindering blood flow. Sufferers experience severe painful episodes, which can require hospital admission. Life expectancy can be 20 to 30 years shorter than in the general population.
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Toby Bakare, 35, from south London, who has sickle cell disease but received a stem cell transplant, said: “It’s a great day, so many people living with sickle cell disease will now have access to a treatment that gets rid of pain [that] is a blight on your life. I’m lucky I had a sister who was a match.
“Not everyone with sickle has that option so gene editing for those people is a huge step forward. It’s been a long road, but having a transplant has transformed my quality of life for the better.”
Mehmet Tunc Onur Sanli, 42, from London, who was diagnosed with sickle cell disease at 11, said: “Not having to go to hospital for regular transfusions or taking medicine any more would be a dream to me – gene therapy could offer that – but there’s still a lot to consider in terms of the side-effects that could come with this treatment and whether it would be the right choice for me.”
Prof Bola Owolabi, the director of the National Healthcare Inequalities Improvement Programme at NHS England, said: “This represents a monumental step forward in the treatment of people with sickle cell disorder, which is a condition that mostly affects people of Black African and Black Caribbean heritage. This groundbreaking therapy, available on the NHS, represents a very real prospect of a cure for this devastating disorder.
“The NHS remains committed to rolling out new, innovative treatments to narrow the gap in healthcare inequalities, and this cutting-edge therapy could give patients facing severe sickle cell disorder the hope of a much brighter future.”
John James OBE, the chief executive of the Sickle Cell Society, said: “We are absolutely thrilled to see this groundbreaking gene therapy treatment available on the NHS from today. The significance of this milestone for the sickle cell community cannot be understated – today’s result will give hope to many and is the result of determined campaigning.
“We remain dedicated to our commitment to advocating for improved treatment options and better outcomes for all those affected by this condition. For now, we take a moment to celebrate this historic milestone – a turning point in the fight against sickle cell that promises to change lives for the better.”
Yasmin Sheikh, the head of policy and public affairs at Anthony Nolan, said: “This groundbreaking decision to fund the UK’s first ever Crispr-based therapy for patients with sickle cell disorder represents a leap forward in the treatment of this debilitating and life-threatening condition.”
Article by:Source: Tobi Thomas Health and inequalities correspondent
