In a major breakthrough for sickle cell patients in England, the National Institute for Health and Care Excellence (NICE) has approved the use of CRISPR gene-editing therapy on the National Health Service (NHS). This decision marks a significant advancement in the treatment of this inherited blood disorder.
Landmark Approval After Initial Rejection
NICE had previously rejected the gene-editing therapy exagamglogene autotemcel (exa-cel) for sickle cell disease in draft guidance released in March. However, after further review, the watchdog has now given the green light for NHS use, following its earlier approval for treating beta thalassemia.
The UK’s medicines regulator had already approved exa-cel in November 2023, but its high cost of £1.6 million (€1.9 million) per treatment raised concerns over NHS accessibility.
Sickle cell disease affects the shape and function of red blood cells, leading to severe pain, anaemia, infections, and other complications. The disorder is most common among individuals of African, Caribbean, Middle Eastern, and South Asian descent.
Until now, treatment options were limited, with existing therapies often causing intolerable side effects, according to NICE. The newly approved therapy will be available to patients aged 12 and older who suffer from severe complications and for whom a stem cell transplant is suitable but no donor is available.
‘Much-Needed Hope’ for Patients
Funmi Dasaolu, a sickle cell patient who contributed to NICE’s decision, welcomed the approval, calling it a turning point in the fight for better treatment.
“The approval of exa-cel today marks a significant shift in the treatment landscape of sickle cell disease in the UK,” she said. “It is the beginning of re-addressing inequalities in care experienced by so many with the condition and provides much-needed hope.”
A Scientific Breakthrough with Challenges Ahead
CRISPR/Cas9 gene editing, the technology behind exa-cel, was first developed by Emmanuelle Charpentier and Jennifer A. Doudna, who won the 2020 Nobel Prize in Chemistry for their discovery.
The exa-cel therapy, also known as Casgevy, works by removing a patient’s blood stem cells, editing them using CRISPR in a lab, and reintroducing them into the body.
While experts hail this as a groundbreaking step, some uncertainties remain. Professor Felicity Gavins, a pharmacologist at Brunel University London, highlighted that while exa-cel offers a potentially curative treatment, it is not a universal cure for all patients.
“It is critical to continue funding research to develop treatments that benefit the broader sickle cell population and address remaining challenges in care,” she said.
To assess long-term effectiveness, safety, and accessibility, NICE will collect patient data as treatment progresses before conducting a re-evaluation of the therapy in the future.
