- The National Health Service (NHS) in England has approved exa-cel, a groundbreaking £1.65 million gene therapy for sickle cell disease
- Exa-cel is designed for patients with severe sickle cell disease who qualify for a stem cell transplant but lack a matched donor
- Nigeria, with the highest number of sickle cell, cases worldwide could greatly benefit from similar treatments
In a groundbreaking development, the National Health Service (NHS) has approved a pioneering £1.65 million treatment for sickle cell disease in England, offering patients a remarkable 96.6% cure rate.
The new therapy is set to revolutionise the management of the inherited blood disorder, providing a potential long-term solution for those living with its painful and life-threatening complications.
The newly approved treatment, exa-cel, is specifically designed for individuals with severe sickle cell disease who are eligible for a stem cell transplant but lack a suitable donor match.
With this innovative therapy, an estimated 50 patients, including older children and adults, are expected to receive life-changing treatment annually in England.
Sickle cell disease affects approximately 17,000 people in England, with around 4,000 believed to be eligible for exa-cel.
Clinical trials have demonstrated that this gene therapy can prevent painful sickle cell crises, which occur when crescent-shaped blood cells block blood vessels, causing severe pain and potential organ damage.
An impressive 96.6% of trial participants experienced a “functional cure,” meaning their symptoms were significantly reduced or eliminated.
Despite facing an initial rejection by the National Institute for Health and Care Excellence (NICE) in March 2023 due to insufficient evidence, exa-cel has now been approved, marking a historic milestone in sickle cell treatment.
“This is a monumental step forward for individuals with sickle cell disease, particularly those of Black African and Black Caribbean heritage,” said Prof. Bola Owolabi, Director of the National Healthcare Inequalities Improvement Programme at NHS England.
Speaking to The Guardian, Owolabi emphasised the NHS’s commitment to addressing healthcare inequalities by introducing groundbreaking therapies that could offer a brighter future for patients battling this condition.
While the approval of exa-cel is a breakthrough for England, it also holds significant implications for countries with high sickle cell prevalence, such as Nigeria.
Nigeria bears the highest burden of sickle cell disease globally, with over 4.3 million people affected and approximately 150,000 babies born with the condition each year.
The country’s healthcare system continues to struggle with the growing challenges of managing the disease, as sickle cell-related complications can reduce life expectancy by 20 to 30 years.
The success of gene therapies like exa-cel could pave the way for increased research, innovation, and accessibility in regions where the disease is most prevalent.
If widely adopted, such treatments could significantly improve the quality of life for millions of patients worldwide.
With the NHS leading the way in adopting cutting-edge treatments, the fight against sickle cell disease has taken a crucial step forward.
The approval of exa-cel offers new hope to patients, signalling a future where sickle cell disease is no longer a lifelong battle but a condition with a viable cure.
US Policy Shift: Nigeria announces investment in health sector
Meanwhile, TheRadar earlier reported that Nigeria announced significant domestic health sector investments following a shift in the United States (U.S.) development assistance policy.
The Federal Executive Council (FEC) approved two major initiatives, aimed at bolstering human capital development and sustaining essential healthcare services across the country.
The approved plans include a $1 billion initiative to enhance human capital development and N4.8 billion for the Presidential Treatment Programme for HIV patients.