The Story:
There has been welcome progress made in recent years in the treatment of sickle cell disease, a group of blood disorders, typically inherited, that manifests in a number of ways: anemia, the swelling of extremities, and strokes among them. Millions of people have this malady worldwide, including an estimated 100 thousand in the United States. It gets its name from the characteristic sickle-shaped blood cells found in the system of patients.
Significance:
There is a wide disparity in the consequences of SCD depending on where one is born. In high income countries, more than 94% of the newborns with SCD will live to be adults. In some low-income countries, on the other hand, fewer than 50% will make it past five years.
The use of hydroxyurea and regular blood transfusions has been shown to have a great effect in mitigating the consequences of SCD. A recent paper in the Indian Journal of Medical Research said that, despite its demonstrated effectiveness, hydroxyurea remains underutilized even in the HICs.
In Pill Form:
Beyond the important issue of symptom mitigation, there is reasonable expectation that a full cure is on the way. The IJMR piece speaks of work on gene editing that could “modify or correct the defective sickle gene,” and that could be ready for regulatory approval within “the next few years.”
