He has new blood.
A Long Island hospital said it had administered life-changing Rifgenia gene therapy treatment and successfully wiped out sickle cell disease from a Laurelton man.
Cohen Children's Medical Center said patient Sebastien Beauzile, 21, was the first New Yorker to receive groundbreaking treatment.
“This is a revision,” said Dr. Jeffrey Lipton, director of pediatric hematology oncology and stem cell transplantation at the center. “Other drugs fix the illness, but this is a treatment… I think this will replace bone marrow transplants in time.”
Buezil had the first crisis of many crises from the extremely severe cases of sickle cells as a four-month-old baby. Since then, he has consistently come and go to the Northwell facility in New Hyde Park, he said.
“In many cases, the pain was 10 out of 10,” he said. “My back felt like someone was pulling it or hanging from it. My breasts felt like someone was sitting on it.”
He couldn't travel without going to the hospital, struggled to get a job, and had to stop going to Queens Community College due to repeated illness issues.
“He was trying several treatments, but none of them worked,” said Dr. Banu Aygun, assistant director of hematology.
“So when gene therapy becomes available when the FDA is approved [in late 2023]Sebastian was the first patient in our minds… so that he could dramatically change his life. ”
Simple cell
Instead of more painful procedures like bone marrow transplants, Lyfgenia produces new healthy genes inserted into parental cells that connect to stem cells in disease-stricken red blood cells, officials said. Aygun explained that the newly approved treatment costs “millions” through insurance.
Buezil had mixed feelings in early 2024 when he was told about options that involve a treatment for nearly a year. He was certainly excited by the opportunity for a bright future, but worried that he would have to go through a week of chemotherapy to clear out his old stem cells.
“I was a little nervous at first, but the doctors told me about it and told me what the side effects would be, and I was like, 'Why not?'
“To be honest, it wasn't that bad.”
For several months, cells were regularly drained from Bozil's blood and sent to the lab for gene insertion.
In late December, they permeated him again within minutes.
“Now he's producing normal adult hemoglobin in his parental cells. You see it in his blood,” Dr. Ayun said. “That's why he doesn't have symptoms related to sickle cell disease.”
For guarantees, Beauzil had to stay in the hospital for another month, saying January 13th was the day when things started and felt like a new guy.
“When I got the cells, it was like an in vitro experience…it's like a second birthday,” he said.
“I'm not in pain anymore. I've been able to do a lot of new things,” added Beuzil, who worked out without any problems and had the first chance of a carefree vacation.
He is also looking to re-enroll in school to pursue a medical career inspired by his own journey.
“I feel that kids who have sickle cells in particular feel that if someone has experienced what they are going through and that they can become role models for them.”
