Sickle cell gene therapy brings hope to Triangle family: 'It is exciting'

Josh Chapin Image
Saturday, December 9, 2023
Sickle cell gene therapy brings hope to Triangle family
Regulators on Friday approved two new gene therapies for sickle cell disease that doctors hope can cure the painful, inherited blood disorder.

DURHAM, N.C. (WTVD) -- Amarre Thorpe sounds like any other 11-year-old kid. He talks about playing with his three siblings and games on his phone.

But because of sickle cell disease, Amarre can't work up the strength to run down the court like his basketball idol Lebron James.

"I felt achy pain in my stomach, some sharp pain in my neck," he said.

Amarre's pain isn't as bad since he got a bone marrow transplant. Turns out his older brother Malachi was a 100% match.

"It was definitely a challenge to do but luckily with some support from family members, we were able to handle that really well to get the transplant done," said Darren, who works at ABC11 as a security guard.

Darren now has his younger daughter Journey to worry about. She also has sickle cell disease which affects about 100,000 people nationwide.

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A majority of those people are in the Black community.

The new gene therapy approved Friday by the FDA won't be for Darren's son Amarre but maybe for his 1-year-old.

"If we can make it so that patients go through this therapy one time, a one-time autotransplant and take their own cells and change the genetics and give it right back to the patient, the patient's not going to reject it," said Dr. Nirmish Shah, a hematologist at Duke University Medical Center. "It is their own stem cells, it's their own bone marrow."

Dr. Shah zoomed with ABC11 from San Diego where the American Society of Hematology was meeting and talking about the new therapy.

"It is exciting," he said. "The goal is we can continue to have great outcomes that we've seen so far and hopefully not have any potential side effects. That is something we're continuing to learn."

Dr. Shah noted the treatment isn't going to be for every patient.

There are some complications including the cost of one of the treatments which is upward of $3 million. You also have to get chemotherapy which can cause infertility and a number of the patients looking to get this are on Medicaid or Medicare.

But Darren is hopeful this is at the very least more awareness about the disease.

"I hope this brings families that have children with sickle cell in line with having an option," he said.

Duke University Medical Center will also be the North Carolina hub for treatment.