Access to gene therapy for sickle cell disease in Africa
Document Type
Artefact
Department
Internal Medicine (East Africa)
Abstract
In 2017, Gene Therapy (GT) was used to cure Sickle Cell Disease (SCD).1 This was a major achievement in a genetic condition that had first been described over 100 years ago and the therapeutic interventions were limited to disease-modifying medicines (Hydroxyurea), exchange blood transfusion (ExBT) and Haematopoietic Stem Cell Transplant (HSCT). With this milestone, individuals with SCD requested access to curative therapy, particularly in countries in Africa where the disease is most prevalent and causes high morbidity and mortality.2 Unfortunately, this milestone was accompanied by a ‘health crisis’ as countries in Africa observed an increase in the number of patients seeking HSCT outside the continent and a ‘financial crisis’, as both curative interventions (HSCT and GT) were associated with a high cost (USD 100,000 to 2 million USD). Stakeholders working in SCD and related disciplines responded to this situation in an integrated manner that included coordination, healthcare, advocacy, research, and training (CHARTA).3 To increase access to therapy, the strategy had to be bold, innovative, and contemporaneous, optimising existing interventions whilst preparing for new therapies with in vivo GT as the holy grail.4 Furthermore, it quickly became apparent that partnerships had to be at the core of the response. The response to the health crisis included the strengthening and establishment of treatment centres and centres of excellence (TC/CoE) where advanced therapy could be provided, with multi-disciplinary team (MDT) meetings with discussions between healthcare providers from all over the world. The MDT meetings help to improve patient outcomes by offering comprehensive, specialised, integrated and coordinated care, between the referring, transplant and treating centres. The response to the financial crisis required advocacy with patients and engagement with policy makers. Advocacy with patients was needed to understand the needs (treatment or cure) and to see how best to support those who opted for curative therapies.5 Engagement with policy and policy makers had to occur at three levels: country, regional (Africa) and global.6 This included outlining and communicating the need to develop capacity for advanced therapy in-country to reduce patients seeking healthcare outside the country, and Africa. Countries in Africa have strengthened specialists’ services, by different financial models e.g. investing public funds or partnering with private sector or philanthropy. We conclude by recommending that research must be at the heart of the responses; ranging from clinical, social, implementation and basic sciences, with an integrated approach, such as CHARTA, that intentionally coordinates partnerships in financing and policy, whilst centering the patient and public community.
Publication (Name of Journal)
EBioMedicine
DOI
https://www.thelancet.com/journals/ebiom/article/PIIS2352-3964(25)00157-4/fulltext
Recommended Citation
Patel, S.,
Makani, J.,
Bukini, D.,
Malangahe, S.,
Arnold, S. D.,
Rifai, A.,
Moshi, G.
(2025). Access to gene therapy for sickle cell disease in Africa. EBioMedicine, 115, 105713.
Available at:
https://ecommons.aku.edu/eastafrica_fhs_mc_intern_med/479