PEOPLE living with sickle cell disorder have praised a decision by NHS Scotland to approve a groundbreaking gene therapy treatment.
After months of deliberation, the Scottish Medicines Consortium ( SMC) has approved the use of exagamglogene autotemcel ( brand name Casgevy) to treat people with the chronic blood disease.
The gene therapy has been accepted for patients who are older than 12 and are able to have a stem cell transplant, but do not have a suitable donor.
Patrick Eneche, who has lived with sickle cell for more than 40 years, said that the SMC’s decision was a “great achievement”.
Eneche said: “There was a lot of excitement this morning. This is good news and a big relief, as someone who has lived with the disorder for 40 years.
“It’s been a challenge to have a very great life, as sickle cell comes with a lot of pain and affects mental health, and you have to spend a lot of time in hospital.
“It is a very good thing, I hope to explore it to see how it benefits us.”
Around 400 people are believed to be living with sickle cell disorder in Scotland, many of whom are of African or Caribbean descent.
According to the charity Anthony Nolan, the genetic disorder affects the shape of red blood cells, which subsequently get stuck in small blood vessels, leading to severe pain and organ damage.
The Casgevy treatment extracts and modifies a patient’s blood stem cells, allowing the cells to produce healthy red blood cells.
Formerly, stem cell transplants from donors were the only curative treatment available for people with sickle cell disorder, but the new treatment opens the door to relief for those who are unable to find a match.
Christine Membi is the project manager of The Hope Project Scotland, a Glasgow- based charity that raises awareness about the disease and advocates for people living with sickle cell disorder.
The group also organises cooking classes, bowling excursions and other activities for community members.
She said that the decision could “change everything” for those with sickle cell disorder in Scotland.
“The lifestyle will change, as will the time spent on hospital visits and the day- to- day challenges,” Membi said. “People do not think about how sickle cell affects the families of people with the disorder.”
She added: “We are happy they actually approved it. It is a big deal. This is something for us and they are actually looking at us.
“It is a good thing that the Scottish Government has approved it for use, but we will continue to make sure people can access it.”
While the on- off gene therapy has a list price of £ 1.65 million per patient, the NHS in England reportedly negotiated a confidential discount when it was approved for use last year. Speaking about the sickle cell community in Scotland, Eneche said: “It’s a close community. We come together and do reviews and meetings, but we also find a way to have leisure time and make things easier.”
Membi added: “We will continue to campaign to let people know. Many people don’t know about sickle cell and how it affects people.”
In a joint press release with Anthony Nolan, Olalekan Oyedepo, who lives with sickle cell disorder and is co- founder and project coordinator at The Hope Project Scotland, added: “We welcome today’s decision by the SMC to approve Casgevy gene therapy for sickle cell disease patients in Scotland … 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.”
