Imagine being three years old and having been admitted to the hospital over 20 times, needing blood transfusions and morphine, with the doctors telling you that, going forward, these will be your main treatment options. This is the harsh reality for 20 million people living with sickle cell disease around the world.
In Europe, the life expectancy for those living with sickle cell is 60 years, while it can be as low as five years in many African countries, making it one of the most devastating conditions that presents in infancy. But Voxeletor, hailed as a drug effective at improving daily functioning in those with sickle cell disease, offers hope on the horizon despite the long road ahead.
Sickle cell disease was named by modern science in 1910 when a doctor in Chicago treated a Grenadian man for pain and anaemia. However, we know the disease has existed for several millennia across Africa, the Middle East and India. It is caused by a change in genes that transforms red blood cells from their regular doughnut (without a hole) shape to a crescent moon shape, leading to a lack of red blood cells able to transport oxygen around the body, painful episodes, and, in severe cases, strokes during childhood or early adulthood.