The National Health Service (NHS) has approved a groundbreaking £1.65 million treatment for sickle cell disease in England, offering patients a promising 96.6% cure rate.
The new therapy, commended as a significant breakthrough, is expected to change the lives of individuals struggling with inherited blood disorders by providing a long-term solution to the painful and life-threatening condition.
The new treatment, exa-cel, is designed for patients with severe sickle cell disease who qualify for a stem cell transplant but do not have a matched donor.
Approximately 50 patients, including older children and adults, are expected to benefit from this innovative therapy
Clinical trials for exa-cel have demonstrated that the therapy can prevent painful sickle cell crises, which occur when blood vessels become blocked. Researchers found a “functional cure” in 96.6% of participants who received the treatment.
Notwithstanding initial rejection by the National Institute for Health and Care Excellence (NICE) last March due to insufficient evidence, the approval now represents a major breakthrough in the treatment of sickle cell disease.
With the National Health Services’ approval of this cutting-edge treatment, the fight against sickle cell disease takes a significant step forward, offering hope for a future where patients no longer have to endure the severe pain and risks associated with the condition.