Sebastien Beauzile’s recently announced cure from sickle cell disease brings hope to thousands of mostly Black Americans who suffer from the rare disease, but the multi-million dollar cost of the gene-editing therapy raises question about access and affordability.
“I’m not in pain anymore. I’ve been able to do a lot of new things,” 21-year-old Beauzile said during a press conference last week.
Doctors at Cohen Children’s Medical Center in Long Island, NY successfully used a newly-approved Lyfgenia gene-editing treatment to cure Beauzile of a painfully debilitating disease he’s endured since he was a baby, the New York Post reported on March 13.
“This is a fix,” Dr. Jeffrey Lipton, the center’s director of pediatric hematology oncology and stem cell transplantation, told the Post. “Other drugs modify the disease, but this is a cure … I suspect this will replace bone marrow transplants in time.”
The nearly-year long procedure that ended in December involved doctors draining Beauzile’s cells, and delivering them to a lab where they were then injected with the Lyfgenia genes. Those genes were then re-inserted back into the body where they connected with Beauzile’s red blood cells.
The price tag for the sickle cell cure, however, is a whopping $3.1 million per treatment for Lyfgenia and $2.2 million for Casgevy, Forbes has reported.
The history behind sickle cell disease
The announcement represents the latest in a handful of successful treatments or cures after the FDA approved gene-editing “cure” for sickle cell disease in 2023.
“Now he’s producing normal adult hemoglobin in his parent cells. You see it in his blood as well,” said Dr. Banu Aygun, associate chief of hematology at the center. “That’s why he’s not having any symptoms related to his sickle cell disease.”
Sickle cell anemia affects 100,000 Americans each year, 90% of whom are Black or African-American, according to the Centers for Disease Control. The disease creates a mutation in red blood cells that become sickle-shaped, making it difficult for all cells to bring oxygen throughout the body.
It comes with excruciating pain that can lead to strokes, heart disease and a life expectancy nearly 20 years shorter than the general population. The disease has its roots in Africa, where the mutation naturally evolved in humans thousands of years ago to fight off Malaria, according to the American Red Cross.
Who can afford new “cure”?
For decades patients and their families have complained about the lack of support at hospitals and the lack of federal funding dedicated to researching a cure for sickle cell disease.
In a previous interview with the Black Wall Street Times, Velvet Brown-Watts, founder and executive director of Supporters of Families with Sickle Cell Disease, explained the only approved drug for sickle cell in the ’80s and ’90s was a repurposed cancer drug.
“When you look at diabetes and cancer, they had how many options,” Brown-Watts said. “And at the time, sickle cell only had one. And it wasn’t even a drug for sickle cell.”
That’s changed in recent years as more treatments have become available. Last week’s announcement of a cure in New York has brought even more hope to patients around the country and around the world, but it’s unclear how many of those patients could afford the it.
The median income for a Black-led household was $54,000 in 2023, meaning half of Black households earn less and half earn more. They would have to afford the insurance, and that insurance policy would have to approve the treatment. Without insurance, it would take the median-level Black household over 50 years to afford a cure for sickle cell disease.
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