Science Summit meeting on Sickle Cell

Introduction

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 and 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.
Objectives
The session will:
• Educate stakeholders on state-of-the-art understanding of the SCD in Africa and major scientific activities underway that have both immediate and long-term implications for patient outcomes.
• Outline how the outputs of basic biomedical science, including the elucidation of genetic underpinnings of disease, will translate to improved patient care.
• Define key implementation research learnings and outstanding needs to assure successful delivery of newborn screening at large

Abstract

This Session will focus on three critical areas of science: (a) Biomedical science to help better understand the nature of SCD in patients in Africa; (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 and interventions; (c) Partnerships in Research and development of new therapeutics . The focus will be on gene-based therapies that are designed to cure SCD.
Muhimbili University of Health & Allied Sciences (MUHAS), Tanzania, will convene this Session. MUHAS is part of SickleinAfrica, a multinational consortium comprising national-level stakeholders in Tanzania, Nigeria, Ghana, Uganda, Zimbabwe, Zambia, and Mali, with coordinating centres in Tanzania and South Africa. MUHAS is also part of Sickle CHARTA, A Consortium for Health, Advocacy, Research and Training fin Africa. The main goal is to reduce the burden of SCD through the generation and application of scientific knowledge, and conducting research in Africa.
Target audience
Multi-disciplinary stakeholders that are working to address SCD at country, regional, and global levels. Participants are anticipated to include patient advocates, clinicians, policy makers & other government officials, researchers, industry, NGOs, philanthropies, and multilateral health organizations.

Expected outcomes

Educate stakeholders on state-of-the-art understanding of the SCD in Africa and major scientific activities underway that have both immediate and long-term implications for patient outcomes.
• Outline how the outputs of basic biomedical science, including the elucidation of genetic underpinnings of disease, will translate to improved patient 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 within Africa