New Sickle Cell Drug Met With Mixed Emotions

The time has come for the U.S. Food and Drug Administration (FDA) to decide on a new sickle-cell treatment. The FDA has until Dec. 8 to decide the fate of the drug. 

As previously reported by BLACK ENTERPRISE, Exa-cel, the one-time gene therapy created by Vertex Pharmaceuticals and CRISPR therapy has the potential to change lives. As people await the FDA’s decision, concerns are raised about the drug’s costs, accessibility, and transparency regarding potential side effects. The sickle cell treatment will be the first of its kind, but its side effects are unknown, according to NBC News. It will be the first drug to use CRISPR, a gene editing tool that alters DNA. 

The novelty of the drug and sentiments of distrust for healthcare held by the Black community may lead to hesitancy to receive the treatment.

Derek Robertson, president of Maryland Sickle Cell Disease Association, has two sons with sickle cell. Robertson said, “We are aware that there are a number of individuals who are still a little bit hesitant. But overall, we’re excited mainly because it’s another option.” 

Robertson, whose wife works in patient advocacy at Vertex, believes the focus should be on establishing a drug that is effective and to “get it to the people who need it.” Later, the focus can turn to finding ways to ensure affordability and access.

As Dr. Cecelia Calhoun, a hematologist-oncologist and the medical director of the Adult Sickle Cell Program at Smilow Cancer Hospital at the Yale New Haven Health System in Connecticut, discussed the disease process she said sickle cell affects the “quality of life and ability to just function as normal humans.”

Kayla Smith Owens, a 25-year-old graduate of Bowie State University, shared her experience living with sickle cell disease. She was first diagnosed at 12 after experiencing mild pain and fatigue during puberty. The symptoms were exacerbated following her matriculation in 2016. 

“I had no college experience at all,” she said. “My time in college consisted of being in the hospital for seven days, coming home, catching up, and then going right back to the hospital.”

Living with the disease drastically altered her college experience. “I couldn’t join clubs. I didn’t have time to make friends because my time was spent in the hospital,” she continued.

After graduating from the university with a degree in criminal justice, Owens said she has not had the opportunity to work because she is constantly in pain and “always sick.” Owens said she feels the new drug “would be huge and change [her] life tremendously.” 

Dr. Glassberg has had some patients who participated in the Exa-cel and Blue-bird clinical trials. Blue-bird is an additional sickle cell drug under FDA review. He said his patients have “normal blood counts,” and they’re also able to run 3 miles a day because “they don’t have sickle cell anymore.”

To receive the treatment, patients must undergo chemotherapy. Dr. Glassberg attributes the risks associated with chemotherapy to why he believes less than 10% of people being treated for sickle cell at Mount Sinai will pursue the drug as treatment. However, he said the risks have “nothing to do with gene therapy.

The cost of the treatment is an additional concern among doctors and patients. The drug could cost up to $2 million, according to the Institute for Clinical and Economic Review.

According to an analysis by NORC, a research organization associated with the University of Chicago, 87% of people who are both enrolled in Medicaid and have been diagnosed with sickle cell disease are Black. 

As far as coverage and insurance costs are concerned, Dr. Glassberg  said “my experience has been nobody really knows what’s going to happen until it just hits the market and then stuff starts to happen,” he said. “Then we sort of figure out how to make it work.”

Dr. Glassberg also acknowledged feeling of mistrust from the Black community for the healthcare industry. With the help of his colleges, Dr. Glassberg said, they have worked to build “foundations of trust and gain confidence” with their patients through support groups and a process where a group of patients assess and “approve” research studies. 

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