UK to roll out ‘revolutionary’ new treatment for sickle cell disease

A new treatment for sickle cell disease has been developed and will soon be offered to patients in the United Kingdom.

According to the UK National Health Service (NHS), the treatment is the first one developed in over 20 years.

Sickle cell disease, mostly common among Africans and Caribbeans, is a disease that causes the red blood cells to become hard, sticky and take a crescent shape.

When the cells travel through small blood vessels, they get stuck and clog the blood flow which causes pain. The sickle cells also die early, which causes a constant shortage of red blood cells.

People with the condition endure severe pain called a ‘sickle cell crisis’ that can occur multiple times per year, which often require hospital visits or admission.

The new drug, known as Crizanlizumab, made by Novartis, is injected into the vein and can be taken alone or alongside standard treatment and regular blood transfusions.

It works by binding to a protein in the blood cells to prevent the restriction of blood and oxygen supply that lead to a sickle cell crisis.

The NHS said the drug will reduce the number of times a sickle cell patient needs to go to accident and emergency (A&E) by “two fifths”.

People aged 16 and over who suffer from multiple sickle cell crises per year will be eligible for the treatment.

UK’s National Institute for Health and Care Excellence (NICE), which recommended the drug be available on the NHS, said it could not recommend the drug for routine use yet owing to high uncertainty about the long-term effectiveness of the treatment and its associated costs.

But the agreement with the NHS will allow patients to access the drug while extra data is collected through clinical trials.

Amanda Pritchard, chief executive of NHS England, said the drug will help as many as 5,000 people over the next three years to have a much better quality of life.

“This is a historic moment for people with sickle cell disease who will be given their first new treatment in over two decades,” she said.

“This revolutionary treatment will help to save lives, allow patients to have a better quality of life and reduce trips to A&E by almost half.”