The Food and Drug Administration has approved a gene therapy that can eliminate complications from sickle cell disease in children as young as 2 years old, expanding access to a treatment previously limited to patients 12 and older.
Rickey Buggs, 13, is among the first patients to receive the treatment at TriStar Centennial Children's Hospital in Nashville.
"For him, sickle cell had been all he knows. He was born with it," said Dr. Lametra Scott, Rickey’s mom.
He’s had to endure bouts of pain for much of his young life.
"One day it will be like somebody is cutting me. Some like a bee kind of stinging me," Rickey said.
However, a new gene therapy treatment just approved by the FDA means many young children won’t have to experience that pain.
Previously, the gene therapy was approved for use in people 12 and older; that’s why Rickey was able to receive it.
This new approval from the FDA means children as young as 2 years old can receive it.
Dr. Haydar Frangoul, director of pediatric cellular therapy at TriStar Centennial Children’s Hospital, said he helped pioneer the treatment.
He said the therapy works by collecting stem cells from a patient's blood, using an electrical process to alter them, and then returning them to the patient.
"We use a technique to gene edit them to make high levels of fetal hemoglobin and put them back into the patients," said Frangoul.
Before this treatment, curing sickle cell disease required finding a compatible donor for a stem cell transplant — something that was not possible for most patients.
"80% do not have somebody who is a match to them," Frangoul said.
The newly expanded approval is significant because early intervention can prevent long-term damage caused by the disease.
"Remember, treating this disease early will prevent the long-term complications from it," Frangoul said.
Frangoul said the breakthrough was something he once considered impossible.
"If you came in and told me we could take someone else's cells out, gene edit them, and give them back to them, I would have said there is no way that can be done, that's science fiction," he said.
Shortly after my interview with Rickey, he and his mom headed home after more than 40 days in the hospital, where he was recovering from having his edited stem cells transplanted back into him.
Rickey’s mom has started an organization to raise awareness about Sickle Cell Disease; click here to find out more about that.
Do you have more information about this story? You can email me at robb.coles@newschannel5.com.
This story was reported on-air by a journalist and has been converted to this platform with the assistance of AI. Our editorial team verifies all reporting on all platforms for fairness and accuracy.
A slip-and-slide for seniors?! Who knew it could stir laughter and tears. Photojournalist Angie Dones captures a story filled with so much joy and one that will tug at your heartstrings.
- Carrie Sharp
