‘Groundbreaking gene therapy’ for sickle cell patients approved for NHS

Campaigners have hailed a “landmark moment” for Scots with sickle cell disorder after a new treatment said to be a “functional cure” for the condition was approved for the use on the NHS.

Stem cell charity Anthony Nolan and the Sickle Cell Society both welcomed the decision by the Scottish Medicines Consortium (SMC) to approve the “groundbreaking gene therapy”.

Approximately 18,500 people in the UK have sickle cell disorder – a genetic disorder that affects the shape of red blood cells, which take on an abnormal sickle shape.

These abnormal cells can get stuck in small blood vessels, leading to blockages that can cause severe pain, organ damage and other complications.

The condition has only been able to cured with a stem cell transplant – but not all patients can find a matching donor.

However, the treatment exagamglogene autotemcel – which is also known as Casgevy – works by modifying a patient’s own blood stem cells, before returning them to their body.

These modified cells then go on to produce healthy red blood cells, addressing the symptoms of sickle cell.

With exagamglogene autotemcel (exa-cel) approved for use on the NHS for patients over the age of 12, Caitlin Farrow, director of strategy and influencing at Anthony Nolan, said: This decision has the potential to be life-changing and underlines the promise of gene therapy for previously difficult to treat or incurable diseases.”

The decision “is a landmark moment for people living with sickle cell disorder in Scotland,” she added.

“Following NICE’s recommendation of Casgevy for use in England last year, it is hugely encouraging to see the SMC take this same step forward, ensuring patients in Scotland can now also benefit.”

Ms Farrow continued: “Treatment options for patients with sickle cell have been limited, with a potential cure only available to those fortunate enough to have a suitable stem cell donor.

“Casgevy offers new hope to many more people, most of whom are from African and African-Caribbean backgrounds, who are living with a disease that has historically been underinvested in.

Paula Shutt, communications manager at the Sickle Cell Society, said: “We are absolutely delighted to see this groundbreaking gene therapy become available to people living with sickle cell in Scotland.

“This marks a significant step forward in ensuring that those affected by sickle cell have access to the care, treatments and support they deserve.”

Sickle cell patient Olalekan Oyedepo, from Glasgow, who co-founded The Hope Project Scotland to help provide support for families affected by sickle cell and other blood disorders, also welcomed the decision.

He said: “This brings Scotland into line with England, where the treatment was approved last year, and represents a significant milestone for patients living with sickle cell.”

“Today’s decision shows what’s possible when health systems recognise the needs of sickle cell patients. Now we must ensure that this recognition extends to comprehensive care provision. Scotland’s sickle cell community deserves nothing less.”