Long Island Boy Makes Gene Therapy History with Treatment for Rare Blood Disorder

A eight-year-old boy from Long Island has become medical history after receiving groundbreaking gene therapy treatment for an extremely rare genetic blood disorder called thalassemia, making him the first patient to undergo this procedure in New York. Yusuf Saeed was born with a condition that causes his body not produce enough hemoglobin, which is crucial component of red blood cells according to the Centers for Disease Control and Prevention (CDC). This resulted in severe anemia from birth.
Dr Jon Fish, who leads stem cell transplantation and therapy at Cohen Children’s Medical Center on Long Island explained that historically there were only two options available: medical management with chronic blood transfusions or a bone marrow transplant for patients suffering from thalassemia. However, Yusuf did not have an appropriate donor to undergo the latter procedure.
Instead, doctors opted for gene therapy treatment which was recently approved and is considered transformative in nature. The process involves collecting blood cells from the patient before sending them off to a lab where they are genetically modified with a new gene that helps produce normal adult hemoglobin. Once ready, these altered cells are infused back into the body of the patient.
Yusuf spent six weeks in hospital during this process and underwent chemotherapy treatment before receiving his newly-modified blood cells. Within just four weeks after being transfused with genetically modified cells, Yusuf’s doctors witnessed improvements thanks to his new therapy that they believe has prevented further need for painful blood transfusions he would normally have received monthly since birth due to thalassemia.
Yusuf will still be required to attend regular medical check-ups every two weeks at present but the hope is that this can eventually reduce down to annual appointments once doctors are completely confident about how successful Yusuf’s therapy has been for his treatment and care in managing symptoms associated with thalassemia.
The gene therapy procedure used on Yusuf could potentially be applied as a preventative, curing or treating measure against other hereditary diseases such as sickle-cell anemia according to doctors at Cohen Children’s Medical Center. The young boy has expressed his gratitude towards the medical team who have helped him so much and he dreams of one day becoming a zookeeper when fully grown up whilst also having a passion for dinosaurs.