NEW YORK — A young Long Island boy has become the first in New York state to receive a new gene therapy for a rare blood disease.
A gene was actually added to the child’s blood cells to cure a life-threatening condition.
Just after he turned 8, Yusef Saeed celebrated another birthday of sorts: a rebirth of his health.
“We are lucky that we were able to get the gene therapy for Yusef so early,” says mother Yusara Ahmed.
The Valley Stream second-grader was born with beta thalessemia, a genetic blood disorder. Because Yusef could not produce red blood cells, he needed transfusions every few weeks.
A bone marrow transplant could have cured him, but there were no matches, not even his twin sister.
“Time passed and we could not find any donor,” said father Farrukh Saeed.
As hopes for a donor faded, another new gene therapy was approved by the Food and Drug Administration. For the first time in New York, a gene was added to Yusef’s stem cells.
“Gene therapy is a transformative treatment. The patient is their own donor. We are essentially collecting the patient’s own stem cells,” says Dr. Banu Aygun, a hematologist at Cohen Children’s Medical Center.
The changed cells are then returned to the patient, a one-time treatment that encourages the bone marrow to do its work.
“He didn’t need a blood transfusion for the first time in his life since mid-February,” said Dr. Jon Fish. “This is a very exciting and transformative moment because this is just the beginning.”
Gene therapy shows promise in similar diseases and eliminates the need for a bone marrow transplant, which Yusef’s aunt needed when she was a child.
“The only option in my sister’s time was to go through a donor, and now it’s really amazing that that’s the only option. It’s mind-boggling,” Ahmed said.
Yusef spoke few words, but made them count.
“Thank you so much to Dr. Aygun and Dr. Fish for taking care of me,” he said.
His parents said they hope he will be a voice of hope.
“One person can help another. That’s how the community grows. So I just hope he becomes that for other children,” Ahmed said.
The doctors’ hope is that Yusef can now live a long, healthy life without ever needing a blood transfusion again.
The first patients to undergo this gene therapy in clinical trials have already completed eight years and are living transfusion-free.
Carolyn Gusoff
