New drug ‘cure’ hope for sickle cell Scots

‘Groundbreaking’ therapy approved by NHS

- BY KATRINE BUSSEY

Crescent-shaped red blood cells affected by sickle cell anaemia and normal cells (round)

NEWS that a drug has been approved for Scots with sickle cell disorder has been described as a “landmark moment”.

The treatment, which is said to be a “functional cure” for the condition, has been given the go-ahead for 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 be cured with a stem cell transplant — but not all patients can find a matching donor.