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Depending on a forthcoming decision by the Food and Drug Administration, a treatment called CRISPR may be the next step toward giving those with sickle cell the hope of a pain-free life.
According to the Associated Press, the FDA was scheduled to start reviewing the gene therapy treatment Nov. 2 for inherited disorders under the sickle cell disease umbrella. The treatment would permanently change DNA in a sickle cell patient’s red blood cells to help their hemoglobin reverse the mutation that causes the disease. The source outlined that after a stem cell retrieval from the blood, the treatment would involve doctors using CRISPR to “knock out the switching gene.”
Next, patients would be put on medications “to kill off other flawed blood-producing cells” and then receive their “altered stem cells.”
The FDA’s approval would make the CRISPR-based gene therapy the first of its kind to be allowed for treatment in the United States. Suppose the governmental agency opts to put it on the market. In that case, it’s reported that a “post-approval” safety study, more research and product labeling about the treatment’s potential risks would all have to be done. Understanding the effects of the treatment seems to be one of the governmental agency’s top priorities.
A decision about the gene therapy treatment is reportedly expected in December.
Notably, potential prices for the treatment haven’t been released, but it’s estimated that “around $2 million would be cost-effective.”
Jarringly, research published in February 2023 outlined that from birth to age 65, women sickle patients spent $1.6 million, and men spent $1.7 million on medical care. The Associated Press referenced the research, but details regarding the sample pool — such as their economic backgrounds — are unclear.
CRISPR stands for “Clustered Regularly Interspaced Short Palindromic Repeats,” according to the National Human Genome Research Institute. The latter source specified that CRISPR is a type of gene technology scientists can use to “selectively modify the DNA of living organisms.” Inventors Emmanuelle Charpentier and Jennifer Doudna received the 2020 Nobel Prize in Chemistry for its discovery.
The Center for Disease Control and Prevention reports that sickle cell affects millions worldwide and is “particularly common” for descendants from sub-Saharan Africa, the Caribbean and other populations.
Last reviewed in July 2023, the CDC’s information stated that sickle cell affects approximately 100,000 Americans. The disease occurs in about 1 of every 365 Black/African American births — and around 1 in 13 of the population’s babies are born with the genetic trait.
Living with sickle cell can be an agonizing life sentence for those born with the disease. Moreover, it can be heart-wrenching for loved ones and caretakers to watch sufferers endure it.
Sickle cell disease is the name of a group of inherited disorders wherein red blood cells are misshapen and sticky instead of being round. “Sickle,” crescent-shaped red blood cells are due to their disordered hemoglobin — which is the protein part of the cell that carries the oxygen.
Red blood cells for those with sickle cell anemia can cause a shortage in the body because they don’t last as long as standard red blood cells do, according to the Mayo Clinic. The result can cause anemia — when the body doesn’t have enough viable red blood cells — and fatigue.
Some of the most characteristic symptoms of sickle cell disease are “episodes of pain,” “pain crises,” or “sickle crises.” The terms refer to the intermittent bouts of pain sufferers of the disorder experience, often in their chest or joints. Mayo Clinic detailed that how often and how long the episodes last depends on the person.
Other symptoms include spleen damage, vision problems and frequent, possibly life-threatening infections. Additionally, sufferers can also experience jaundice, bodily swelling and more.
While there is no cure for sickle cell, current treatments include medications to help with the symptoms and or blood transfusions. Additionally, the Associated Press noted that bone marrow transplants can serve as a “permanent solution.”
Since the disease is inherited, knowing your sickle cell trait status and your partner’s is essential before bringing life into the world.
Learn more, find resources and build community via the Sickle Cell Disease Association of America.
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