NHS Approves £1.65 Million Sickle Cell Treatment With 96.6% Success Rate

The National Health Service (NHS) approves a £1.65 million gene therapy for sickle cell disease in England, offering a potential cure with a 96.6% success rate.

This newly approved treatment, exa-cel, provides a breakthrough solution for individuals suffering from severe sickle cell disease, particularly those eligible for a stem cell transplant but lacking a matched donor. The therapy aims to prevent painful sickle cell crises and significantly improve patients’ quality of life.

NHS estimates indicate that approximately 17,000 people in England live with sickle cell disease, with around 4,000 qualifying for the new therapy. Each year, about 50 patients, including older children and adults, are expected to receive the treatment.

Clinical trials show that exa-cel effectively prevents vaso-occlusive crises by stopping the blockage of blood vessels. The trials report a “functional cure” in 96.6% of participants who undergo the therapy.

The approval follows an initial rejection by the National Institute for Health and Care Excellence (NICE) in March 2023 due to concerns over insufficient evidence. Now, with stronger clinical data, NHS moves forward with offering the treatment to eligible patients.

“This marks a significant advancement in treating sickle cell disease, which predominantly affects individuals of Black African and Black Caribbean descent,” says Professor Bola Owolabi, Director of the National Healthcare Inequalities Improvement Programme at NHS England. “Making this therapy available through NHS brings real hope of a long-term solution for those living with this devastating condition.”

Owolabi emphasizes the importance of expanding access to innovative treatments, stating that this approval reflects the NHS’s commitment to addressing healthcare disparities.

Sickle cell disease remains a major health challenge worldwide, particularly in Nigeria, where the highest number of cases is recorded. Over 4.3 million Nigerians live with the condition, and an estimated 150,000 newborns are diagnosed each year.

The disease alters red blood cells, causing blockages in blood vessels that lead to severe pain, organ damage, and reduced life expectancy by 20 to 30 years. The success of exa-cel highlights the potential for gene therapy to revolutionize treatment in regions where sickle cell disease is more prevalent.

The approval of exa-cel signals a major step forward in global sickle cell treatment efforts. As healthcare systems explore wider adoption of gene therapy, the development sets a precedent for increasing accessibility to life-changing treatments, particularly in high-burden regions.

With this advancement, patients living with sickle cell disease gain new hope for a future free from the chronic pain and complications associated with the condition.