Boy with rare blood disorder receives new life-changing gene therapy treatment

Yusuf Saeed is smiling behind his mask, and he has every reason to. 

The 8-year-old was born with a rare genetic blood disorder called thalassemia. This means his body does not produce enough hemoglobin, a vital component of red blood cells, according to the Centers for Disease Control and Prevention.

As a result, he suffered from severe anemia.

"Historically, the only treatments were medical management with chronic transfusions or a bone marrow transplant," said Dr. Jon Fish, head of Stem Cell Transplant and Cellular Therapy at Cohen Children’s Medical Center in New Hyde Park, Long Island.

Yusuf, despite having a twin sister, didn’t have a suitable donor for a transplant. So instead, doctors opted for newly approved and transformative gene therapy treatment.

Dr. Banu Aygun, the associate chief of Hematology at Cohen, said doctors actually collect the patient's own blood cells to begin this procedure. 

"[We] send them to a lab where the gene is added into the blood cells so the patient starts producing normal adult hemoglobin," she said. "When the cells are ready, we infuse them back to the patient." 

Yusuf was the first patient in New York to receive this infusion. He spent six weeks in the hospital. In that time, he underwent chemotherapy to make room for new cells. Once the genetically altered cells were ready, they were transfused. Within weeks, they started to work on their own. 

Yusuf has to go to the doctor every two weeks. The hope is to transition to monthly and then annual visits.

Doctors hope to use this gene treatment to prevent, treat or cure certain hereditary diseases, including sickle-cell anemia. As for Yusuf – who loves dinosaurs and says he wants to be a zookeeper when he grows up – says "thank you" to the doctors and nurses who made such a difference in his life.