Sickle Cell Disease: Will the latest life-changing treatment reach the people who need it most?
Each year, an estimated 300,000 children are born with sickle cell disease, the majority of them in Africa, but also in India, the Middle East and Mediterranean countries. Where the need is greatest, treatment options are most limited.
- €15,100 Budget in Euros
- 2021 Final release date
- 6 Round winner
- 3 Locations
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Sickle cell disease is a genetic blood disorder that causes red blood cells to become sickle-shaped, rather than their usual donut form. These block capillaries, causing excruciatingly painful vaso-occlusive crises that may last for days at a time, and putting sufferers at risk of organ damage, strokes, necrosis and other serious complications. Upwards of 5 million people are affected, with the majority being in Sub-Saharan Africa, the Middle East and North Africa, and India. Migration is changing this epidemiology, however, and consequently the incidence of the disease is rising in Europe.
In LDCs with a high prevalence of sickle cell disease — Togo, for example — management options are extremely limited, and even strong pain medication to ease sufferers’ crises is hard to come by. It is estimated that 80% of children born with sickle cell disease in the region do not survive beyond their fifth birthday.
In Tunisia, by comparison, the state is able to provide sickle cell disease patients with blood transfusions to improve their ratio of healthy to unhealthy blood cells, and hydroxyurea, a chemotherapy medication that improves the blood’s ability to carry oxygen. These are not a cure, but improve patients’ quality of life and life expectancy. Advances have also been made with bone marrow transplants, but these are complex, costly and infeasible for widespread treatment.
In Germany, at least 3000 people are living with sickle cell disease; 150 000 to 300 000 children are born with it in Africa every year. Yet ironically it is in wealthy countries like Germany where the greatest hope of a cure for SCA can now be found: research involving the use of gene editing technologies has produced promising results. But will such treatments ever reach the places where they are needed most?
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