United States: The National Health Service has received authorization to provide a “groundbreaking” 1.65m Euros treatment that could potentially cure sickle cell disease patients.
More about the news
The British medical community praised the official approval of exagamglogene autotemcel or exa-cel therapy due to its ability to edit the faulty gene in stem cells derived from a patient’s body.
The NHS believes these funds will provide this advanced treatment annually to 50 suitable patients aged ten and above who suffer from sickle cell disease and lack donor matches.
Exa-cel demonstrated promising results in clinical trials because it stopped sickle cell crises from harming patients, according to research data, which showed treatment success in 96.6% of patients who got exa-cel.
Widely prevalent condition
Health and Care Excellence denied approval for the treatment in March 2022 because they needed additional information about treatment effectiveness, the Guardian reported.
National Health Service reports that sickle cell disease affects people of African or Caribbean descent most often.
The population of England includes approximately 17,000 people diagnosed with Sickle cell disease.
Out of the existing 4,000 eligible candidates, 3,000 have received approval for the new medical therapy.
People with sickle cell disease develop crescent-shaped blood cells, which obstruct proper blood circulation.
People with sickle cell disease need to be hospitalized when suffering from intense painful episodes.
The average lifespan of individuals with sickle cell disease amounts to less than twenty to thirty years of existence compared to the general population.
What more are the experts stating?
According to John James OBE, the chief executive of the Sickle Cell Society, “We are absolutely thrilled to see this groundbreaking gene therapy treatment available on the NHS from today. The significance of this milestone for the sickle cell community cannot be understated – today’s result will give hope to many and is the result of determined campaigning,” the Guardian reported.
“We remain dedicated to our commitment to advocating for improved treatment options and better outcomes for all those affected by this condition. For now, we take a moment to celebrate this historic milestone – a turning point in the fight against sickle cell that promises to change lives for the better,” he added.
Furthermore, Yasmin Sheikh, the head of policy and public affairs at Anthony Nolan, added, “This groundbreaking decision to fund the UK’s first ever Crispr-based therapy for patients with sickle cell disorder represents a leap forward in the treatment of this debilitating and life-threatening condition.”