The U.S. Food and Drug Administration has approved two treatments for sickle cell disease that the medical community ignored because most of the patients are Black and because there was little money for treatment.

Sickle cell is an inherited blood disorder affecting more than 100,000 in the U.S. Most of those afflicted are African American and Hispanic American, and a few are White. 

Sickle cell disease is caused by a mutation in hemoglobin, a protein found in red blood cells that delivers oxygen to the body’s tissues.“

“These approvals represent an important medical advance with the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. 

The FDA-approved therapies Casgevy and Lyfgenia will be used for sickle cell for men and women 12 years old and older with recurrent vaso-occlusive crises. 

A vaso-occlusive crisis occurs when the microcirculation is obstructed by sickled RBCs, causing ischemic injury to the organ supplied and resultant pain.

Lyfgenia is a cell-based gene therapy that uses a lentiviral vector for genetic modification. 

With Lyfgenia, the blood stems are genetically modified to produce HbA, a gene therapy derived from functions similar to red blood cells with HbA, which have a lower risk of sickling and occluding blood flow. 

Sickle disease has a long history of being a neglected disease due to systemic racism in medical care, research, and funding.