New gene-editing treatment for sickle cell could be great news in Central Florida

The Food and Drug Administration on Friday approved a gene-editing therapy that’s seen success in treating the most severe form of the disease.

CRISPR Therapeutics had the treatment, brand name Casgevy, approved in the United Kingdom last month after seeing a successful global trial in which 28 out of 29 patients reported being free of severe pain.

The treatment is a breakthrough against sickle cell — which previously had only one known cure: a bone marrow transplant — and could make a difference for many lives in Florida. The Sunshine State boasts a yearly average of about 7,000 new cases a year, twice that of the national average, according to a 2023 report by the Florida Agency for Health Care Administration.

“Sickle cell disease is a common genetic disorder in certain ethnicities. The African American population is No. 1, but we are seeing a lot of other ethnicities such as Asian and patients from Iran and Iraq,” said Dr. Asad Sheikh, medical director at the Orlando Heath Cancer Institute. “Florida is quite a hodgepodge of all these ethnicities.”

South Florida and Central Florida lead the state’s average diagnosis per year, according to the report.

Sickle cell causes a shortage of red blood cells and a lack of hemoglobin, which grabs oxygen molecules and binds them to cells. As a result, those with sickle cell experience chronic pain.

According to CRISPR, Casgevy uses a patient’s bone marrow to edit a gene to express a type of hemoglobin.

“What Casgevy does is increase the amount of fetal hemoglobin in the body,” Sheikh said.

The gene-editing treatment won CRISPR the Nobel Prize for chemistry in 2020. While impactful, the treatment may not have an immediate impact in the Central Florida area and its large pool of patients, Sheihk said.

“It’s going to take up to several years for the average sickle cell patient to have access to this type of highly advanced therapy,” he said.

The first limitation is the expense. Casgevy is going for a high price point of $1 million, according to Health Education England’s Genomics Education Programme. Other outlets have reported the treatment can cost $2 million.

According to a report by the American Medical Association, 70% of patients identify themselves as disabled. Additionally, low employment rates have also been reported along with that disabled status. The report concludes these patients are likely to have lower earnings.

Additionally, a transplant center is required to administer Casgevy. Only five in the state as listed by the National Alliance of Sickle Cell Centers. Orlando Health is not listed but has a transplant center, Sheihk said, but far more will be necessary to make the treatment accessible.

“We need to create more transplant centers in Central Florida. And on top of that, we need to employ a lot of manpower for this type of gene editing therapy,” Sheikh said.

Even with the limitations Casgevy has in accessibility, Sheikh still sees the FDA approval as a huge first step in eradicating sickle cell.

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