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HomeNewsResearchers Make Breakthrough Discovery of Cure for Sickle Cell Disease and Get...

Researchers Make Breakthrough Discovery of Cure for Sickle Cell Disease and Get FDA Approval 

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In a historic first, the Food and Drug Administration (FDA) approved an effective treatment for sickle cell disease last week.

Clipart of DNA strands
Source: NIH Image Gallery/Flickr

Sickle cell disease is a medical condition that affects patients’ livelihoods. In the US, demographic studies posit that over 100,000 Americans are suffering from the illness.

The game-changer for sickle cell patients is the CRISPR gene editing tool. Emmanuelle Charpentier and Jennifer Doudna are the brains behind the CRISPR gene editing tool, which won them a Nobel prize in 2020. CRISPR Therapeutics has perfected the gene-editing method’s accuracy and deployed it to cure sickle cell disease.

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CRISPR Therapeutics and Vertex Pharmaceuticals are the joint developers of the first-ever gene-editing treatment regime for sickle cell disease. They called the therapy Casgevy. The sickle cell disease is the product of an anomaly in the red blood cell. Usually, red blood cells have a disc-like shape.

On the contrary, red blood cells of sickle cell patients take a sickle or crescent shape. This results in the clumping together of blood cells, blood clotting, and finally blocking of blood vessels. Such biological disorders come with several life-altering and life-threatening symptoms. Summarily, sickle cell patients are unable to lead everyday lives.

To cure sickle cell disease, the Casgevy treatment involves editing the patient’s stem cells. The treatment can stop the future production of sickle blood cells by editing the stem cells. Stem cells are responsible for producing new red blood cells and are found in the bone marrow.

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The Casgevy treatment is a one-off but may take several months and stages. First, the patient has a series of blood transfusions for about four months. Afterward, their stem cells are extracted for gene editing at a laboratory.

In the meantime, the patient will then go through chemotherapy to kill off any stem cells or bone marrow still present in the patient after the extraction. Finally, the patients undergo a re-infusion of the edited stem cells, then some months to recovery.

Of course, the Casgevy treatment is not a walk in the park. It is costly and may come with complications like temporal hair loss, nausea, skin color changes, etc. However, if successful, sickle cell patients get to enjoy an everyday life like any healthy human.

The price tag of Casgevy is $2.2 million per individual and does not include the cost of preliminary and convalescence treatments for the patient. Only sickle cell patients aged 12 years and above can undergo the Casgevy treatment.

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Before gaining FDA approval, Casgevy therapy underwent clinical trials to prove its efficacy. Starting in late 2020, 46 patients agreed to participate in the clinical trials. Of that figure, the treatment was successful in 29 of the patients.

Of course, there are still doubts about the long-term effects of Casgevy. However, most patients with successful treatments already live healthy and everyday lives. 

Interestingly, Casgevy is not the only gene-editing treatment for sickle cell in recent times. Last week, the FDA approved a sickle cell treatment called Lyfgenia, developed by Bluebird Bio.

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