The Food and Drug Administration (FDA) has approved the first ever Sickle Cell Cure in the United States to mark a breakthrough of the disease.
According to reports, Sickle Cell affects over 10,000 people in America alone majority of which are black. This breakthrough will go a long way in reducing the fatalities caused by the disease.
Reports indicate that the Sickle Cell cure will be in form of therapy known as Casgevy. This was developed from Vertex Pharmaceuticals and CRISPR Therapeutics. It is the first medicine to be approved in the United States that uses the gene-editing tool CRISPR.
According to Dr. Alexis Thompson, chief of the division of hematology at Children’s Hospital of Philadelphia, its impressive how the gene editing tool is a game changer since its discovery to now inventing Sickle Cell Cure.
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“I think this is a pivotal moment in the field. It’s been really remarkable how quickly we went from the actual discovery of CRISPR, the awarding of a Nobel Prize, and now actually seeing it being an approved product.”
Although Casgevy was approved for people ages 12 and older, it removes the need for a donor. Using CRISPR, it edits the DNA found in a patient’s stem cells to remove the gene that causes the disease.
Casgevy works by editing the DNA in a patient’s stem cells, which are responsible for making the body’s blood cells. After that, they no longer produce sickle-shaped cells.
However the therapy has been described as expensive. With treatment alone having to require $2.2 million per patient. A figure that doesn’t include the patients care in the hospital or chemotherapy and other treatment that come with the cure. Therefore, it will be very expensive for most families to afford the procedure alone.
The clinical trial included 46 people in the U.S. and abroad, 30 of whom had at least 18 months of follow-up care after the treatment. Of those, the treatment has been successful in 29 patients.