6 days Ago By [email protected] (Shannon Miller, Katie Oborn)
For those battling sickle cell disease, pain and fear are tragically routine, yet a significant scientific advancement offers newfound optimism for the UK's 17,500 affected individuals. A groundbreaking £1.6 million gene-editing therapy is set to be provided to those most in need, potentially acting as a 'functional cure'.
The NHS gave the green light for the pioneering gene therapy treatment exagamglogene autotemcel on January 31, targeting those who qualify for stem cell transplants but lack a compatible donor. Sickle cell represents the UK's quickest expanding genetic disorder, characterised by abnormally sickle-shaped red blood cells which result in excruciating blockages that can damage organs or cause severe complications such as blindness or even a fatal stroke. Approximately 50 people every year across the UK will receive this one-off therapy, involving lab-based modification of their blood stem cells to induce greater foetal haemoglobin production in red blood cells.
Patients will first undergo intensive chemotherapy to purge their bone marrow of defective cells before receiving their transformed cells back. But Calvin Campbell, an NHS worker from London with six decades of living with sickle cell disease, voices concerns about the remedy reaching only a select few: "People start to believe 'I'm not going to have to go through this anymore' and they're told 'sorry, you're not even going to be considered'," he told the Mirror . Calvin, 60, said: "People need to stop using the word cure.
The vast majority of people with sickle cell won't be affected by it, you have to be unwell but not too unwell. But for the people who are eligible, it will most definitely help." Having lived with the condition since he was just six months old, Calvin describes the agony he endures during a sickle cell 'crisis' – the term used for these attacks.
With triggers ranging from weather changes to stress and dehydration, the debilitating pain experienced by those with sickle cell can persist for days or even weeks, generally necessitating hospital treatment and strong painkillers such as opioids. "It is akin to someone taking a baseball bat, smashing and breaking every single bone in my body then forcing me to run a marathon as they continue to beat me, and after all that I still have to get on with life while they carry on beating me," Calvin explains. Calvin is a senior community engagement officer for NHS Blood and Transplant (NHSBT), whose role is to specifically get Black and Brown communities to donate blood and organs.
He also sings a choir called B Positive, made up of people whose lives are affected by sickle cell disease, which was created by the NHS to spread awareness and bring people together. As a child, he temporarily lost his sight to retinopathy and later suffered severe sickle cell leg ulcers, a complication that left him in pain for over 10 years. It was so severe he lost all his skin and most of the flesh from below his knees down to the soles of his feet.
Reflecting on those harrowing times, he said: "You could have put your arm through the space in my leg, the doctor described my pain as akin to being set on fire. And that wasn't the worst, I ended up paralysed for a couple of years. No one has ever explained why.
" Another heart-wrenching ordeal occurred when Calvin's brother passed away at a tragically young age. Upon receiving wreaths at his door, he discovered, to his shock, that they bore his own name - mourners had assumed that Calvin had died, because he was always so unwell. Today, Calvin's routine includes monthly blood exchanges, a treatment that has allowed him to travel for leisure recently- a first for him.
He described the crucial support he requires, which takes "roughly 128 to 132 people a year" to keep him alive - adding that this shows "how big the need is". His dedicated consultant haematologist, Dr Emma Draser, remarked on the vast improvement in her patient's quality of life: "What stands out about Calvin is his persistence and determination to succeed despite his severe sickle cell disorder and its acute and long term impacts on his health. "Calvin is always on the go.
He is able to travel now, has an active family life with his grandchildren and extended family, not to mention the work he does for the NHSBT, our local patient support group and our network patient public voice group. I am always telling him to slow down." Regarding the new therapy, she expressed optimism, in that the new gene therapy gives hope.
Even though it isn't appropriate for all people with sickle cell disorder, it "increases choice". New trial medications will continue to be licensed - and give people options for "improved outcomes in sickle cell disorder". Currently, many patients like Calvin depend on frequent transfusions, yet finding a compatible match is challenging, often leading to extended intervals between exchanges or the administration of fewer blood units.
One donor making a significant impact is Amino Ali, a renal nurse with the RO blood type - the most suitable match for those with sickle cell, which minimises the risk of negative reactions. Amino, 29, from Oxford, discovered her rare blood type following her initial donation and said she felt "really special" when she was told, especially knowing that one donation "could save up to three lives". Amino said: "It's just a small gesture to do something greater.
I've been encouraging my family and my friends and two of my friends have started donating blood and my siblings. I know that there are misconceptions that exist around donating blood, especially in the Black community, I do want to try and break the misconceptions because you are essentially helping out your own community and you're not losing anything by donating." The Mirror recently visited the new Brixton Donation Centre, a pivotal development for boosting Black blood donors, as Lambeth boasts one of the most significant Black demographics in London.
It's noted that some 55 percent of Black individuals in London possess the RO blood type, while just 2 percent of people from other ethnic groups share this type. Upon arriving in the centre's reception area, visitors are greeted by the inspiring 'hero wall' showcasing donors. The venue is designed to provide an enjoyable experience, from comfortable seating for donations to tempting local treats mirroring the essence of the community, such as rum cake.
As of January 2025, nearly 33 percent of all RO blood donations across England and Wales were sourced from south London donor hubs, spotlighting the importance of this specific site in raising numbers among Black blood donors. Marikany Keita, 20, made his first donation at the centre after witnessing his friends grapple with illness. Marikany said: "I want to help, I see it everywhere and know by donating my blood it will help people.
I saw this (Brixton Donation Centre) last week and I booked it the exact same day. If I didn't see this I wouldn't have been here today. No way.
" What is sickle cell disease? Sickle cell disease affects the shape of red blood cells, causing them to form into a sickle or crescent shape. This abnormal shape leads to the cells getting jammed in blood vessels, which can result in severe pain or even cause deadly long-term complications, such as organ failure and strokes. Predominantly found in individuals of African or Caribbean heritage, sickle cell disease is among the fastest-growing genetic disorders in the UK, compelling many patients to undergo regular blood transfusions for survival.
Symptoms can begin as early as six months old. The NHS lists the most common symptoms as painful episodes, susceptibility to infections, and anaemia. Gene therapy, blood exchanges and transfusions, hydroxycarbamide (also known as hydroxyurea), along with stem cell or bone marrow transplants, are some of the treatments available.
How to donate: The demand for blood donations for sickle cell treatment has surged by 67% in the last half-decade. The NHS is making an appeal for more Black blood donors to step forward. In the UK, cases are on a sharp rise, with the demand for blood donations to treat the condition having surged by 52% in the past five years.
The condition primarily impacts African and Caribbean communities, but it can affect anyone. For information about your nearest centre and how you can contribute, visit www.blood.
co.uk.
