A cutting-edge gene therapy has been approved for NHS use for some patients with severe sickle cell disease.
Casgevy, also known as exa-cel, was the first treatment to be licensed using gene-editing tool Crispr, which earned its inventors the Nobel Prize for chemistry in 2020.
It works by editing the faulty gene in a patient’s own stem cells.
NHS officials estimate that around 50 people a year will receive treatment now that it has been approved for use for certain patients with severe sickle cell disease.
Here patients have reacted to the news:
– Funmi Dasaolu
University student Funmi Dasaolu has experienced chronic fatigue and pain her entire life.
The 31-year-old, from Oxfordshire, has been admitted to hospital many times due to the condition – in 2022 she was admitted to hospital seven times.
For the last five years she has been receiving regular blood transfusions to help with her symptoms.
“Today is a momentous day for those living with or affected by sickle cell disorder,” she said.
“After months of campaigning, I’m overjoyed and so very grateful exa-cel has been finally approved.
“It will be truly transformative for patients and offers us the chance of a life without this terrible condition. A chance to grow old, to fulfil our dreams and to live a pain-free life.”
– Toby Bakare
TV producer Toby Bakare said that as a child he frequently missed school due to painful sickle cell crises and was admitted to hospital four to six times a year.
He received a stem cell transplant from one of his siblings.
On the approval of the treatment, the 35-year-old, from south London, said: “This decision is a game changer for the thousands of people like me living with this disorder.
“They now have a chance to live without pain, fatigue and all the other symptoms of sickle cell which can make quality of life so poor.”
– Mehmet Tunc Onur Sanli
Mehmet Tunc Onur Sanli was diagnosed with sickle cell disease when he was 11.
The 42-year-old, from London, said: “Because of my illness, I often experience pain in my chest, bones and muscles.
“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.
“I also suffer from regular sickle cell crises. Last year, I had to go to the hospital at midnight after waking up in severe pain, and overall I had to visit the hospital five or six times due to crises.
“The pain is the worst I have ever felt in my life – it’s hard to put into words.
“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.”
– Lanre Ogundimu
Sickle cell patient Lanre Ogundimu described how the condition has affected her.
She said: “In 2018, I suffered a stroke, pulmonary embolism and a blood transfusion reaction, which led to 10 days in an intensive care unit, 30 days in hospital, six months in physiotherapy, over 12 months in therapy and nine months not working.
“I felt weak all the time. This was the lowest point in my life.
“It impacted my freedom and independence, my income, my career trajectory, and my ability to contribute towards society.”
