UK approves gene-editing drug for sickle cell disease

Medical regulators in the UK have approved a gene therapy that aims to cure two blood disorders.

The treatment for sickle cell disease and beta thalassemia is the first to be licensed using the gene-editing tool known as Crispr, a revolutionary advance for two inherited blood conditions, both triggered by errors in the gene for haemoglobin.

Sufferers of sickle cell disease produce unusually shaped red blood cells that can cause problems because they do not live as long as healthy blood cells and can block blood vessels, causing pain and life-threatening infections.

Sufferers of beta thalassemia do not produce enough haemoglobin, which is used by red blood cells to carry oxygen around the body, often resulting in the need for a blood transfusion every few weeks of their lives.

In a laboratory, the gene-editing tool Crispr uses molecular scissors to make precise cuts in the DNA of the cells, thus disabling the faulty gene.  The modified cells are infused back, allowing the body to start producing functioning haemoglobin.

In trials, 28 out of 29 sickle cell patients were free of severe pain and 39 of 42 beta thalassemia patients no longer needed blood transfusions for at least 12 months.

Professor Josu de la Fuente, who is leading the UK trials in adults and children, said “This is the kind of technology you read in science fiction books and you never thought it would be reality and here we are in my professional life being able to be part of the story and deliver it to patients.”

Currently around 15,000 people in the UK have sickle cell disease, most with a Caribbean or an African family background.  Around 300 babies are born in the UK with sickle cell disease each year.

More than 1,000 people in the UK are affected by thalassemia, mainly those of southeast Asian, Middle Eastern and Mediterranean origin.

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Posted by Karen Motley, Clinical Negligence Department, Chadwick Lawrence LLP (jacquelinevance@chadlaw.co.uk), medical negligence lawyers and clinical negligence solicitors in Huddersfield, Leeds, Wakefield and Halifax, West Yorkshire.

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