FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease
U.S. Food and Drug AdministrationDec 08, 2023
FDA Approves First Gene-Editing Treatment for Sickle Cell Disease
The Facts
The US Food and Drug Administration (FDA) on Friday approved a breakthrough treatment for patients suffering from sickle cell disease, a painful ailment that affects roughly 100K people across the US, primarily people of color. Sickle cell disease makes red blood cells bend into inflexible sickle shapes, thus preventing the normal delivery of oxygen in the body and causing bone deterioration, organ failure, and strokes.
The FDA approved two treatments: one called Lyfgenia from the company Bluebird Bio, and the other Casgevy by partners Vertex Pharmaceuticals and CRISPR Therapeutics, both of which use patients' own blood cells and are approved for people 12 and older. The Casgevy therapy works by removing a patient's cells from their bone marrow, editing the genes, and then infusing the body with billions of modified cells.
The Spin
Narrative A
These treatments are a medical marvel that will help better the lives of roughly 100K African Americans as well as Hispanic Americans across the country over time. With the FDA's approval, doctors will now be allowed to take a patient's own blood and replace the faulty red blood cells with new, genetically edited healthy ones, thus bringing long-awaited relief from the horrific pain and fatigue typically associated with this disease.
Narrative B
While the use of gene editing is remarkably good news for those with diseases like sickle cell, the public should also have a seat at the table when debating the potential harms of this new medical tool. For example, as voluntary procedures like the newly approved treatment are widely seen as positive, potential future uses like creating desirable traits in babies are seen by many as unethical. It should also be noted that this treatment has not been documented as a "cure" just yet.
Nerd narrative
There's a 62% chance that an inorganic nanoparticle-enabled cancer therapy will be approved by the FDA before 2031, according to the Metaculus prediction community.