Scientists Finally Discover 'Potential Cure' for Sickle Cell Disease
- The NHS has approved a groundbreaking £1.65 million gene therapy, exa-cel, offering a potential cure for sickle cell disease
- Around 50 eligible patients annually in England, primarily of African and Caribbean heritage, will benefit from this one-time treatment
- Campaigners and patients celebrate the milestone, calling it a transformative step in reducing healthcare inequalities
A revolutionary gene therapy, offering a potential cure for sickle cell disease, has been approved for use on the National Health Service (NHS) in England.
The £1.65m treatment, known as exagamglogene autotemcel (exa-cel), is a one-time gene therapy that edits the faulty gene in a patient's own stem cells.
Source: Getty Images
This breakthrough comes as a significant relief to the approximately 17,000 people living with sickle cell disease in England, with around 4,000 believed to be eligible for the new treatment.
SCD affects millions worldwide
Sickle cell disease is a genetic disorder that affects the shape of blood cells, hindering blood flow and causing severe painful episodes.
The approval of exa-cel is a monumental step forward in the treatment of sickle cell disease, which disproportionately affects people of Black African and Black Caribbean heritage.
Nigeria, with an estimated 5-6 million carriers, is considered the epicenter of the disease worldwide.
New treatment offers hope
Clinical trials have shown that exa-cel can stop painful sickle cell crises, with researchers finding a "functional cure" in 96.6% of participants who received the treatment.
The NHS estimates that around 50 patients per year will receive the cutting-edge treatment, which is expected to significantly improve their quality of life.
Campaigners and healthcare professionals have welcomed the news, hailing it as a historic milestone in the fight against sickle cell disease.
Prof Bola Owolabi, Director of the National Healthcare Inequalities Improvement Programme at NHS England, said:
"This groundbreaking therapy, available on the NHS, represents a very real prospect of a cure for this devastating disorder."
John James OBE, Chief Executive of the Sickle Cell Society, added:
"We are absolutely thrilled to see this groundbreaking gene therapy treatment available on the NHS... 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."
While the approval of exa-cel is a significant breakthrough, some patients have expressed caution, citing concerns about potential side effects.
Mehmet Tunc Onur Sanli, a 42-year-old sickle cell patient from London, said:
"Not having to go to hospital for regular transfusions or taking medicine anymore would be a dream to me – gene therapy could offer that – but there's still a lot to consider."
Nigerian doctor forced to operate own daughter
Earlier, Legit.ng reported that Dr. Amos Akinbiyi, a Nigerian-born obstetrician-gynecologist based in Canada, had taken legal action against the Saskatchewan Health Authority (SHA) and its employees, alleging breach of contract, intimidation, conspiracy, and defamation.
The lawsuit follows a series of incidents dating back to 2017 that, according to Dr. Akinbiyi, highlight systemic racism and unethical treatment within the health system.
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Source: Legit.ng
