He’s got new blood.
A Long Island hospital said it administered life-changing Lyfgenia gene therapy treatment and successfully wiped away sickle cell disease from a Laurelton man.
Cohen Children’s Medical Center said patient Sebastien Beauzile, 21, is the first New Yorker to have received the breakthrough treatment, The Post was first to report.
“This is a fix,” said Dr. Jeffrey Lipton, the center’s director of pediatric hematology oncology and stem cell transplantation. “Other drugs modify the disease, but this is a cure … I suspect this will replace bone marrow transplants in time.”
Beauzile, had his first of many crises from his extremely severe case of sickle cell as a 4-month-old baby. Since then, he has consistently been in and out of the Northwell facility in New Hyde Park, he said.
“Lots of times, the pain was 10 out of 10,” he said. “My back felt like somebody was either pulling on it or hanging on it, my chest would feel like somebody is sitting on it.”
He couldn’t travel without going to a hospital, had a hard time working a job, and had to stop going to Queens Community College because of recurring issues with the disease, which made his life substantially challenging.
“Although he had tried several treatments, none of them were working,” Dr. Banu Aygun, associate chief of hematology, said.
“So when gene therapy became available when it was FDA approved [in late 2023], Sebastien was the first patient in our minds…so that he can dramatically change his life.”
An easy cell
Lyfgenia creates a new, healthy gene inserted into parent cells that connect to the stem cells of red blood cells afflicted by the illness instead of more painful procedures like bone marrow transplants, officials said. Aygun explained that the newly approved treatment costs “millions” through insurance.
Beauzile had mixed emotions in early 2024 when told about the option that would take close to a year of treatments. He was certainly excited about a chance for a brighter future but was anxious about needing to go through a week of chemotherapy to clear out his old stem cells.
“At first, it was a little nerve-wracking. But the doctors spoke to me about it, told me what the side effects would be, and I was like, ‘why not?’” Beauzile, who passed time in the hospital building Lego and playing “Dragon Ball Z” video games, said.
“Honestly, it wasn’t that bad.”
For months, cells were periodically drained from Beauzile’s blood and sent to a lab for the gene insertion.
In late December, they were successfully re-infused back into him in a matter of minutes.
“Now he’s producing normal adult hemoglobin in his parent cells. You see it in his blood as well,” Dr. Aygun said. “That’s why he’s not having any symptoms related to his sickle cell disease.”
For assurances, Beauzile had to stay in the hospital for about another month and said that Jan. 13 was the day he felt like a new man as things kicked in.
“When I got my cells, it was like an out-of-body experience…like a second birthday,” he said.
“I’m not in pain anymore. I’ve been able to do a lot of new things,” Beauzile added of working out with no problems and having the first chance for a carefree vacation.
He is also looking to re-enroll in school to pursue a medical career, inspired by his own journey.
“Especially with children who have sickle cell, I feel like if they had somebody that experienced what they are experiencing, went through what they go through, then I could be that role model.”
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