Sickle cell patients share delight over approval of new treatment

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.

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.

“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.

“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

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.”