New sickle cell gene therapy treatments give hope to North Carolina families

The family of a Spring Lake 9-year-old said their daughter has shown improvements following her participation in a clinical trial for sickle cell disease gene therapy.

Leilani Beasley has more energy since undergoing the treatment, which is one of the new therapies already approved by the FDA for patients 12 and older.

"It does include chemotherapy to eliminate the natural stem cells that come into the hospital to get that process. They would then get an infusion of their own stem cells back that have been modified and then their body will recover from that chemotherapy, from that infusion or the delivery of the new cells until they start to take over and do their job," said Dr. Jennifer Rothman, the Director of the Pediatric Sickle Cell Program at Duke.

Sickle cell disease impacts about 100,000 Americans, about 90% of whom are Black. The disease is hereditary; Leilani's father Carlton also has it.

"You have to be born with it, and you live the rest of your life with it," Carlton said.

The disease's life-altering symptoms include anemia, extreme pain, jaundice and stroke. While Beasley takes medicine daily to manage his symptoms, he and his wife Tamara were interested in other treatment options for Leilani.

"After seeing what she's went through, seeing what he went through, we talked about it. It was like, we don't want that for her," Tamara said.

That's when they learned of a clinical trial at Duke, which was searching for patients of Leilani's age.

"It changes the way the red cells are made so they no longer sickle, which means they no longer get trapped in the organs, they no longer get trapped in the bones," Rothman said.

So far, the results have been promising.

"Individuals who have gone through gene therapy, through these clinical trials have had a near normalization of their blood counts, near normalization of their physical functioning and reduction in pain, reduced hospitalizations, improvements overall in their in their quality and function in life," Rothman said.

The Beasleys have been encouraged by the results, as they continue to stick together to support Leilani, even shaving their heads in solidarity as she underwent chemotherapy.

"With this breakthrough in treatment, it gives her the opportunity to have what I consider a normal life," Tamara said.

Clinical trials are still ongoing for patients younger than 12.

"It was definitely eye-opening to see the advancements from when I was a child," Carlton said.