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FDA approves new sickle cell treatments

By Tia McKenzie Dec 11, 2023 | 4:22 PM

JACKSON, Miss. (WJTV) – For the 100,000 patients living with sickle cell disease in the United States, there is hope when it comes to finding a cure. The U.S. Food and Drug Administration (FD) recently approved gene therapies for the inherited blood disorder.

Jeanne Tate, the chairperson for the Mississippi Sickle Cell Foundation, lives with the disease that afflicts mostly Black people in the U.S.

“With having sickle cell disease, it just doesn’t affect your blood. It affects your entire body,” she said.

Even though the FDA approved two gene therapies to treat patients, some concerns have been raised.

“With the price, who’s covered? You know, will Medicaid cover this? Will insurance be able to cover? How would the patients… this be funded for the patients? And a second thing is also the length of time they would also take for a person to be able to get this treatment, because first you have to do the chemotherapy,” said Tate.

The disease occurs in one out of every 365 African American births, according to the Centers for Disease Control and Prevention (CDC).

“The health care system for sickle cell disease has been great, not just in Mississippi, but across these states. We don’t get the best care sometimes at the hospital, especially when we have to go to these emergency rooms, and we’re waiting six to eight hours just to be seen by a doctor,” Tate stated.

Through the therapy technology CRISPR, cells are removed from the body, edited in a lab and returned to the patient. Until now, a bone marrow transplant from a donor was the only way to treat sickle cell disease.

“In my world, I want to see some immediate… I want to see an immediate cure now that can benefit all patients. That’s what I’m looking for,” said Tate.

The FDA approved the gene therapy for those 12 and up.