Achieving the Sustainable Development Goals (SDGs) for health in many countries in Africa will include addressing sickle cell disease (SCD), a chronic non-communicable disorder (NCD). Africa is the epicenter of the disease burden: an estimated 80% of patients live in Africa and more than 300,000 new babies with SCD are born on the continent each year. Historically, health systems in many regions have been ill-equipped to provide optimal preventative and therapeutic healthcare over the life-course of patients with SCD. The result has been high rates of mortality and severe morbidity beginning in early childhood. In this context, the current era of scientific innovation holds promise for helping patients, health workers, policy makers, and other stakeholders to make a sustained impact on population health as it relates to SCD.
This Thematic Session will focus on three critical areas of scientific contributions: (a) Biomedical science to help better understand the nature of SCD in patients in Africa specifically, with an emphasis on discovering genetic underpinnings of disease pathophysiology; (b) Implementation research to guide the effective delivery of interventions proven to save lives from SCD in Africa including newborn screening and distribution of existing medicines; (c) Partnerships in research and development of new therapeutics with target product profiles that will facilitate wide and sustained use in Africa. The focus will be on gene therapy to cure SCD.
Muhimbili University of Health & Allied Sciences (MUHAS), Tanzania, will convene this Thematic Session. MUHAS is part of SickleInAfrica, a multinational consortium comprising national-level stakeholders in Ghana, Mali, Nigeria, Tanzania, Uganda, Zambia and Zimbabwe, with coordinating centers in South Africa and Tanzania. MUHAS also hosts Sickle CHARTA, a consortium for health, advocacy, research and training in Africa for SCD. The main goal is to reduce the burden of SCD through the generation and application of scientific knowledge, and by conducting research in Africa.
The event speakers will feature national and global leaders in SCD-focused biomedical research, implementation science, and R&D. The event will serve to educate a diverse audience of stakeholders on current research efforts underway and to foster dialogue that will inform strategies for accelerating SCD-focused science, promoting the development of research infrastructure through partnerships with global partners, and assuring the sustainability of science in improving health of SCD in Africa
The session will:
- Educate stakeholders on a state-of-the-art understanding of SCD in Africa and major scientific activities underway that have both immediate and long-term implications for health outcomes.
- Outline how the output of basic biomedical science, including elucidating genetic underpinnings of disease, will translate to improved health care.
- Define key implementation research learnings and outstanding needs to assure successful delivery of newborn screening at large scale in Africa and the safe and effective distribution of approved medicines such as hydroxyurea that are proven to save lives and reduce severe morbidities.
- Showcase current efforts to discover definitive gene therapy cures for SCD and explore the anticipated enablers and barriers to achieving widespread access for patients in Africa.
- Recommend pragmatic approaches to cross-sector collaboration that will maximize the quantity and quality of research to address SCD in Africa with an emphasis on conducting research in Africa.
