Stem cell charity Anthony Nolan and the Sickle Cell Society welcome the decision from the SMC (Scottish Medicines Consortium) to recommend the CRISPR gene therapy, exa-cel (exagamglogene autotemcel), as a treatment for people with severe sickle cell disorder in Scotland.
Approximately 18,500 people in the UK have sickle cell disorder. It is 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.
Exa-cel, also known by the brand name Casgevy, involves extracting and modifying a patient’s own blood stem cells before returning them to their body. These modified cells will then produce healthy red blood cells, addressing the symptoms of sickle cell.
Historically the only curative treatment available to people with sickle cell in Scotland was a donor stem cell transplant – however not all patients have a matching donor available. This decision by the SMC now means that Casgevy could provide a ‘functional cure’ for anyone unable to have a stem cell transplant in Scotland.
Caitlin Farrow, director of strategy and influencing at Anthony Nolan, said: “This is a landmark moment for people living with sickle cell disorder in Scotland. 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 from the UK’s first CRISPR-based therapy."
“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.”
“This decision has the potential to be life-changing and underlines the promise of gene therapy for previously difficult to treat or incurable diseases.”
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.”
“It is a particularly important moment for Scotland to see a treatment based on Nobel Prize-winning science being applied to a debilitating condition that has, for far too long, had limited treatment options - and to see that innovation has been manufactured, amongst other places, right here in Edinburgh.”
“While we celebrate this milestone, we know there is still much more to do as sickle cell remains the fastest growing genetic condition in Scotland. We will continue to campaign for greater access to treatments, improved care pathways, and sustained investment, so that everyone living with sickle cell can achieve the best possible quality of life.”
Glasgow based Olalekan Oyedepo, who lives with sickle cell and is co-founder and project coordinator at The Hope Project Scotland, said: “We welcome today’s decision by the SMC to approve Casgevy gene therapy for sickle cell disease patients in Scotland. 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.”
Anthony Nolan will continue to advocate for the needs of cell therapy patients and work to ensure everyone can access the treatments they need.
To find out more about Anthony Nolan, the stem cell register and how the charity supports people with sickle cell disorder, visit: www.anthonynolan.org