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Last Wednesday, Kendric Cromer, a 12-year-old Black patient from the suburbs of Washington, D.C., became the first person in the world to receive a groundbreaking gene therapy treatment that may cure sickle cell disease (SCD).
According to the New York Times (NYT), Cromer began the first step of his extensive treatment process at the Children’s National Hospital in Washington on Wednesday. Bluebird — one of two companies given authorization by the Food and Drug Administration (FDA) to create a gene therapy method for sickle cell — administered the process. According to the outlet, doctors removed parts of Cromer’s bone marrow stem cells that will be “genetically modified” in a specialized lab for his treatment.
The process will take months to complete. Bluebird will need to collect hundreds of millions of stem cells from Cromer, a grueling procedure that will take up to six to eight hours each session.
Once a sufficient quantity of stem cells has been collected, they will be transported to Bluebird’s laboratory in Allendale, New Jersey. Skilled technicians will infuse hemoglobin genes to replace the mutated ones responsible for the 12-year-old’s sickle cell disease.
After three months, the modified cells will be returned, providing Kendric with red blood cells that won’t deform into fragile crescent shapes, reducing the risk of blockages in his blood vessels and organs.
SCD is a genetic blood disorder characterized by abnormal hemoglobin, the protein in red blood cells that carries oxygen throughout the body. In individuals with SCD, the red blood cells become rigid and sickle-shaped instead of flexible and round. These sickle-shaped cells can stick together and block blood flow, leading to pain, organ damage, and other complications.
Sickle cell disease disproportionately affects Black people, particularly those of African descent.
The Centers For Disease Control and Prevention (CDC) notes that SCD is prevalent among populations from regions where malaria is or was endemic, such as sub-Saharan Africa. In the US, SCD affects approximately 1 out of every 365 Black or African-American births. Additionally, about 1 in 13 Black or African-American babies is born with sickle cell trait, a condition in which a child inherits the sickle cell gene mutation from one parent.
The groundbreaking procedure could help millions of Black people living with the disease, but it will be some time before the gene processor is readily accessible to the public. Even though integrating a new gene into stem cells can be accomplished within a few days, the comprehensive testing for purity, potency, and safety typically spans several weeks. Technicians must nurture the cells in the laboratory before commencing these assessments.
The extensive nature of the process means that Bluebird can only treat the cells of approximately 85 to 105 patients each year. The cost of the procedure must also be reduced significantly. Currently, Bluebird has set the price of its gene therapy, Lyfgenia, at $3.1 million, rendering it one of the most expensive treatments available, NYT noted.
SEE ALSO:
Sickle Cell Awareness: North Texas Brothers Seek Black Donors With Start Of Nonprofit
Howard University Receives Grant To Focus On Sickle Cell Disease Education
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