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A new cure for sickle cell disease is underway

The center for disease controls, CDC, defines sickle cell disease as “a group of inherited red blood cell disorders.” Sickle cell is rare, and it disproportionately impacts African Americans. About 100,000 people in the US are diagnosed with sickle cell and, of those, 20,000 have what’s considered severe disease.

Red blood cells contain hemoglobin, which is a protein responsible for carrying oxygen, a very important necessity of life. A normal and healthy red blood cell is round and it easily moves through tiny blood vessels to carry the previously mentioned oxygen to all parts of the body. A very crucial job, without it, we would not live life at all. For someone who has Sickle Cell disease, the hemoglobin is not normal, which leads the red blood cells to become unnaturally dense and sticky, looking more like the letter ‘C’ than a healthy round shape. 

The sickle cells die almost instantly, living a very short life, which causes a constant shortage of red blood cells. This causes pain,other serious symptoms such as infection, acute chest syndrome and stroke.

According to CNN, a new cure may be under way for those suffering from Sickle Cell Disease. A cutting-edge therapy is currently under review that offers the sickle cell community hope for a cure, a rare thing in the world of those with Sickle Cell Disease. CNN reported that, “An independent committee is helping the FDA think through how it should evaluate a treatment called exa-cel that could potentially cure people of sickle cell disease, a painful and deadly disease with no universally successful treatment.” 

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If these studies and research prove significant, a major breakthrough could soon be around the corner and on the horizon. “The FDA said treatment for severe sickle cell is an ‘unmet medical need.’”

The studies contain genetic editing, to hopefully find a cure, and with this kind of genetic editing, scientists could inadvertently make a change to a patient’s DNA. The FDA reportedly  “wanted the experts’ advice so it could understand what criteria it should use to evaluate the treatment and determine how to evaluate long-term safety issues.” 

An FDA presentation to the panel suggested the agency may have some questions about the data. Saying that the data presented lacked confirmatory testing, calling it “concerning.” Also it was noted the study’s size was too small to see how accurate the data could really be. With much going on in this study, and as it is still a developing study, many updates have yet to be announced that could greatly affect the progress of this prospective cure for those with Sickle Cell Disease. The main point to take away is that the FDA is expected to make an approval decision by December 8, 2023.

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Katherine Anderson, Editor-in-Chief
Katherine Anderson is in her senior year at Hart High School and has been part of the Smoke Signal since her sophomore year. Anderson is excited about serving as the Editor In Chief this year and looks forward to making the most out of her last year! When not at school, you can find her with friends, family, dancing, traveling the world, or staring at a sunset.
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