The FDA approves a treatment for sickle cell disease. The article states that this approval marks the first of two potential breakthroughs for inherited blood disorders.
Casgevy, a therapy from Vertex Pharmaceutical and CRISPR Therapeutics, was approved for people ages 12 and older, using CRISPR, which edits the DNA that is found in the patient's stem cells to remove the gene that causes the disease. According to the CDC, in someone who has sickle cell disease, the hemoglobin is abnormal, causing the red blood cells to look like a "C-shaped farm tool", which is called sickle. Sickle cells can travel through small blood vessels which leads to them getting stuck and clog the blood flow, which leads to health problems. Casgevy edits the DNA in patient's stem cells, which are responsible for making the body's blood cells so that they no longer produce sickle-shaped cells. This one-time treatment requires multiple steps that take months before the patient gets the modified stem cells. "It begins with a series of blood transfusions over three to four months, after which the stem cells are extracted from the patient's bone marrow and sent off to a lab where they are edited. Hannah said. Before they can be reinfused into the patient, however, doctors need to make sure no flawed stem cells remain in the body, To do so, chemotherapy is used to destroy the patient's bone marrow. Only then can be edited stem be reinfused into the patient, followed by another month or two in the hospital to allow the cells to grow and the patient to recover". The long-term effects are still unclear, but patients will be monitored for any signs for the next years. The cost of this treatment, however, is expensive and many patients are not able to afford it.
I came across this article because I was interested to know how the treatment works and its effect on patients. I was truly amazed reading this article. The approval of this treatment brings hope to many people who have sickle cell disease. I do hope that this treatment is made affordable so that everyone can receive it. Knowing that a cure for sickle cell disease was made, also brings hope that in the future many more treatments will be approved for other genetic diseases.