CHARLOTTE, N.C. — A recipient of sickle cell gene therapy says the treatment has changed her life. 

What You Need To Know

  • The FDA approved two cell-based gene therapy treatments for sickle cell disease

  • A woman who did a sickle cell gene therapy clinical trial says it changed her life

  • The gene therapy costs $2.2 million

"I haven't had a pain crisis in like a year," said Bria Blount. "I can travel. I can get jobs now."

The FDA has approved the first gene therapy to treat people with the blood disorder. Prior to the approval, a bone marrow transplant was the only cure for sickle cell disease.

Blount's mom, Antowanna Carpenter, says Blount was diagnosed with sickle cell at 2 months old. It's a mutation in hemoglobin, a protein in the blood, that causes red blood cells to be in a crescent shape and is most common in African Americans.

"These sickled red blood cells restrict the flow in blood vessels and limit oxygen delivery to the body’s tissues, leading to severe pain and organ damage," according to a news release from the FDA. "The recurrence of these events or crises can lead to life-threatening disabilities and/or early death."

"The moment that I found out, I literally hit the floor," Carpenter said. "I could only imagine the battle that we were going to be up against."

Blount, a North Carolina native, says she lacked the same type of energy her classmates had in middle and high school. She had her first pain crisis in 2016 when she was across the country in California for college.

"It is like the most crucial pain that a lot of people or anybody could, like, endure," Blount said.

She described it to her mom as feeling like all her bones are breaking at the same time. For four years, Blount says she was in and out of hospitals in California because of pain crises that started out being three months apart, then one month, then weeks apart. She says the doctors she saw on the West Coast were uneducated about sickle cell, not believing her severe pain, and offering short term solutions.

"People were not listening to me. They had discriminated because of my skin color, that I was just out seeking drugs. They would not help me whatsoever, and it came to the point where I just didn't want to go to the hospital anymore," Blount said.

Blount moved back to Charlotte, North Carolina in 2020 where she says doctors knew more about how to treat sickle cell. Almost immediately, she was enrolled in a clinical trial for cell-based gene therapy based at Cincinatti's Children's Hospital.

For six hours straight, five days in a row, medical staff extracted cells from her body. About 11 months later, her modified cells were ready. With her new cells, Blount says it has been about a year since she had a pain crisis.

"I can really be able to do things and not worry about, 'Am I going to be sick or hospitalized?'" Blount said.

While the gene therapy has been a relief for Blount, the treatment remains out of reach for many people who have the disease. According to the American Family Physician Journal, it costs $2.2 million for a single course of treatment. The medical journal Blood Advances notes, the lifetime burden of medical costs for people with sickle cell averages $1.7 million.

Blount says as more people struggle with sickle cell and fight to find a solution that works well for them, more professionals need to be educated on the topic.

"There are doctors out there that do not know how to treat sickle cell disease, and it affects our lives," Blount said." It affects our families' lives because there is no protocol. There is no protocol for parents, and that is why we are here today to advocate for that."