NICE approves sickle cell gene therapy
A ground-breaking sickle cell treatment has been approved by NICE (National Institute for Health and Care Excellence)—signifying a huge step towards better care and quality of life for sickle cell warriors.
ACLT and other organisations have been campaigning for a moment like this. NICE’s approval for its use on the NHS is a real ‘milestone’. As a cohort of people living with sickle cell in the UK now have the chance to treat the underlying cause of their condition, after having gone too long without the option of a potential lifesaving treatment.
Over 17,000 people in the UK are affected by this condition, primarily impacting individuals of Black African and Caribbean heritage. The condition is caused by a genetic change in haemoglobin (a key protein in red blood cells), resulting in red blood cells becoming sickle-shaped. These sickle-shaped cells do not live as long as healthy red blood cells, and they can clump together as they travel through blood vessels, reducing oxygen supply to vital parts of the body. This can put people at a risk of organ damage, stroke, heart failure and causing crisis that largely reduces their quality of life.
"This approval is a ground breaking milestone in the journey of better care for those living with sickle cell."
Casgevy is a gene editing therapy treatment made by Vertex Pharmaceuticals for sickle cell disorder and beta thalassemia. It’s the first approved therapy to use CRISPR technology. It involves modifying the patient’s own stem cells to produce healthy red blood cells, preventing them from becoming sickle-shaped. Patients must undergo ‘conditioning’ chemotherapy to prepare their bodies to accept the edited stem cells. This approach can provide a ‘functional cure’ for individuals who are unable to undergo a stem cell transplant.
Frequently asked questions:
It is a multi-step process that can involve long stays in hospital.
The patient’s stem cells are extracted (since it is the stem cells in their bone marrow that produce the diseased haemoglobin, which causes red blood cells to become sickle-shaped). These stem cells are then genetically modified using a gene-editing technology called CRISPR, which can pinpoint the exact moment when foetal haemoglobin switches to its adult form, resulting in the sickle cells. The genetically engineered stem cells are then returned to the patient, enabling them to produce healthy foetal haemoglobin and normal red blood cells.
Older children and adults with a severe form of sickle cell disease (with multiple crises) who are eligible for a stem cell transplant, but do not have an available matching donor.
For more information about the sickle cell gene therapy visit; NICE approves groundbreaking one-off gene therapy for severe sickle cell disease | NICE
ACLT WEBINAR
New Sickle Cell Gene Editing Therapy: Q&A
Sickle Cell Warriors, Clinicians, and ACLT explore the advancements in the new gene editing therapy for sickle cell warriors.
After the approval of gene therapy treatment for sickle cell warriors, we hosted a webinar exploring these advancements and what they would mean for those seeking this treatment. This event brought together leading experts, researchers, patients (warriors), donors, and healthcare professionals to discuss the latest innovations in CRISPR (pronounced “crisper”), an acronym for Clustered Regularly Interspaced Short Palindromic Repeats. CRISPR is a technology that allows scientists to alter the DNA of living organisms, offering transformative potential for individuals affected by SCD. It is based on a bacterial defence system that protects against viruses.
Clinicians and speakers:
Dr Rachel Kesse-Adu – Consultant Haematologist specialising in sickle cell disorder.
Dr Steven Okoli – Consultant Haematologist and Director of Sickle Cell Alliance.
Dr Dale Seviar – Consultant Haematologist at Guy’s and St Thomas Hospital.
Professor Mark Layton – Clinical Director of the North West London Sickle Cell and Thalassaemia Network.
Kye Gbangbola – Sickle cell warrior – Longest serving chair of the Sickle Cell Society.
Anderson Massamba – Sickle cell warrior and recipient of a stem cell transplant.
Olaolu Baikie – Sickle cell Warrior, previously worked with the NHSBT to raise awareness on sickle cell and blood donation.
Hayley King – Founder of Cianna’s Smile Charity, supporting families impacted by sickle cell.
Pastor Ian Sweeney – TED Field Secretary, Seventh Day Adventist Church
