Sickle cell disease places a heavy burden on many African countries. The World Health Organization reports about 515,000 babies are born with the condition each year, with around 80 per cent of cases in Sub-Saharan Africa. There the disease is a leading cause of death among young children, a situation that health officials and advocates say reflects late diagnosis and limited treatment access.
On 9 February the Ugandan Ministry of Health introduced a policy that requires all babies born in health facilities to be screened for sickle cell disease at birth, free of charge. Official figures estimate about 20,000 Ugandan children are born with the condition each year, and up to 80 per cent die before age five.
In high-income countries regulators have approved new gene therapies. The US Food and Drug Administration approved Casgevy (exagamglogene autotemcel) for patients aged 12 years and over in December 2023 and described it as a milestone; the UK regulator approved the therapy weeks earlier and it was authorised for use in the NHS in England in January. The FDA also approved Lyfgenia, a cell-based gene therapy made by Bluebird Bio, but it carries a warning about possible cancer risks. Casgevy uses CRISPR to edit a patient’s blood stem cells in the laboratory; the edited cells are reinfused after chemotherapy and clinical trials showed a dramatic reduction in severe pain episodes for most treated patients.
Cost remains the main barrier. Casgevy is priced at around US$2.2 million in the United States. In Nigeria an estimated 150,000 babies are born with sickle cell disease each year; bone marrow transplant centres are few and costs range from US$50,000 to over US$200,000. Trust and donor match rates also limit access, and some families who can afford treatment still travel abroad.
Researchers and advocates call for investment in prevention, wider screening and building local capacity. As Lois Bayigga noted, high prices reflect complex technology and specialised facilities, and she said that if viral vectors and gene therapy components were manufactured locally, pricing would drop significantly. Others urge training, technology transfer and stronger government support so treatments become realistic and equitable for low- and middle-income countries.
- Prevention and screening at birth
- Local manufacture of therapy components
- Training and technology transfer
- Stronger government support
Difficult words
- screen — test newborns for a medical conditionscreened, screening
- diagnosis — identification of a disease or medical condition
- access — ability to obtain medical care or services
- regulator — an official body that approves medicinesregulators
- gene therapy — treatment that changes genes to treat diseasegene therapies
- CRISPR — a tool to edit genes in cells
- viral vector — a virus used to deliver genetic materialviral vectors
- equitable — fair and providing equal treatment or opportunity
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Discussion questions
- How could wider screening at birth change outcomes for children with sickle cell disease in low-income countries? Give reasons.
- What are the possible benefits and challenges of manufacturing viral vectors and other therapy components locally?
- What steps should governments take to make advanced treatments more realistic and equitable for low- and middle-income countries?
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