In a historic move, the National Health Service (NHS) in England will now offer CRISPR gene-editing therapy for sickle cell disease (SCD). The National Institute for Health and Care Excellence (NICE) approved the treatment, giving new hope to patients with severe complications.
NICE Changes Course on Exa-cel for SCD
NICE initially rejected the gene-editing therapy exagamglogene autotemcel (exa-cel) in March 2023, citing high costs. However, after further review, the agency approved it for NHS use. Exa-cel had already received approval for treating beta thalassemia.
The UK’s medicines regulator granted its first approval for exa-cel in November 2023. But its £1.6 million (€1.9 million) price tag raised concerns about affordability.
Sickle cell disease changes the shape of red blood cells, leading to pain, anaemia, infections, and life-threatening complications. It primarily affects people of African, Caribbean, Middle Eastern, and South Asian descent.
Until now, treatment options were very limited, often causing severe side effects. The new therapy will be available to patients aged 12 and older who have severe complications and lack a stem cell donor.
A Breakthrough for Sickle Cell Patients
Sickle cell patient Funmi Dasaolu, who helped NICE make its decision, called the approval a huge step forward.
“The approval of exa-cel today is a breakthrough for sickle cell treatment in the UK,” she said. “It also helps address care inequalities and brings hope to patients.”
How CRISPR Therapy Works
The CRISPR/Cas9 gene-editing tool, developed by Emmanuelle Charpentier and Jennifer A. Doudna, won the 2020 Nobel Prize in Chemistry.
Exa-cel, also called Casgevy, uses CRISPR to edit a patient’s blood stem cells in a lab. Doctors remove the stem cells, edit them, and then reinfuse them to fix the genetic defect that causes SCD.
Challenges and Future Research
Experts praise the treatment, but some uncertainties remain.
Professor Felicity Gavins, a pharmacologist at Brunel University London, called exa-cel a major breakthrough but warned it isn’t a cure for all patients.
“We need more research to develop better treatments and improve care for all sickle cell patients,” she said.
To track long-term safety and health effectiveness, NICE will collect patient data and reassess the therapy in the future.