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The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the majority of whom are Black.
The treatment for sickle cell anemia implicitly involves hematopoietic stem cell transplantation (HSCT). This entails replacing the dysfunctional stem cells in the case with healthy bones from a well-matched donor.
The FDA is reviewing a new drug for sickle cell disease that uses CRISPR to edit DNA. Black patients with the disease are excited and hopeful about the treatment.
Voxelotor, sold under the brand name Oxbryta, is a medication used for the treatment of sickle cell disease. Voxelotor is the first hemoglobin oxygen-affinity modulator. Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell patients.
The only cure for painful sickle cell disease today is a bone marrow transplant. But soon there may be a new cure that attacks the disorder at its genetic source.
Sickle cell trait describes a condition in which a person has one abnormal allele of the hemoglobin beta gene (is heterozygous), but does not display the severe symptoms of sickle cell disease that occur in a person who has two copies of that allele (is homozygous).
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