A gene-editing therapy for sickle cell disease, priced at £1.65m, is set to be offered to NHS patients in England.
Around 50 people a year with the inherited blood disorder are expected to receive the one-off treatment, Casgevy, also known as Exa-cel. NHS England’s Prof Bola Owolabi called it a “monumental step forward” and said the one-off treatment holds a real prospect of a cure. A confidential agreement has been made with manufacturer Vertex on the NHS’s payment. Sickle cell disease, mainly affecting people of Black African and Black Caribbean heritage, can be life-threatening and cause intense pain.
A therapy that tweaks a gene to produce healthier red blood cells has been shown to avoid hospital stays for patients with sickle cell disorder. The therapy, which is being studied by NHS chief executive Amanda Pritchard, could be transformative, allowing patients to live free from the fear of sickle cell crises. Nurse Asiawu Imam, who lives with the condition, believes the therapy will be a life-changing moment for many patients. The therapy is expected to provide hope and a sense of community support for those with sickle cell disorders.
How the gene therapy works
Casgevy is a blood cell transplant procedure that involves removing blood stem cells from a patient’s bone marrow and using a gene-editing tool called Crispr to pinpoint specific genes. Instead of directly editing faulty genes, Crispr dampens the switch that produces adult red blood cells in babies. Patients undergo “conditioning” chemotherapy to prepare their bodies for the edited stem cells. Modified stem cells are then transfused back into the body, multiplying and increasing red cell production. However, full treatment requires careful consideration due to potential hospital stays and side effects like headaches and bleeding.
The Sickle Cell Society has announced that a gene therapy treatment called Casgevy will be available on the NHS to people aged 12 and over with recurrent sickle cell crises who cannot find a donor for a stem cell transplant. The therapy has already been approved for transfusion-dependent beta thalassemia and is being given to patients in France, Germany, Italy, and Wales. The Sickle Cell Society’s chief executive, John James, expressed excitement about the new treatment, but acknowledged that not everyone with sickle cell will be eligible for the potentially life-changing benefits.
Source: Graphic Online
