Long Island boy with rare blood disorder undergoes gene therapy

LONG ISLAND, N.Y. (PIX11) -- Until now, the best treatment for some diseases has been bone marrow transplants, a procedure that requires a donor who is a match for the patient. But now, there is a revolutionary new procedure that doesn't require a donor. It actually uses the patient's own cells. The Cohen Children's Medical [...]

LONG ISLAND, N.Y. (PIX11) -- Until now, the best treatment for some diseases has been bone marrow transplants, a procedure that requires a donor who is a match for the patient.

But now, there is a revolutionary new procedure that doesn't require a donor. It actually uses the patient's own cells.

The Cohen Children's Medical Center on Long Island says it has successfully used the treatment on an eight-year-old boy.

Yusuf Saeed was born with a rare blood disorder known as beta thalassemia which reduces the production of hemoglobin. For years he was treated with bone marrow transplants. But the Cohen Children's Medical Center doctors say a major milestone was reached by using the patient's cells for treatment.

Dr. Banu Aygun, the Associate Chief of Hematology explains, "The patient becomes their own donor. We collect the patient's own cells and send them to lab where a gene is added so patient can begin normal life."

Doctors say there is no risk of rejection with the new therapy and that the future looks more promising for young Yusuf. Dr. Jon Fish, Head of Stem Cell Transplant Therapy, notes, "Yusuf will never need another blood transfusion in his life despite the fact that he's needed it to this point."

The new therapy, once it's widely accepted, would no longer require the need to find a donor. Instead, doctors would be able to alter affected cells and infuse them back into a patient.

Yusuf's family understands the pain of bone marrow transplants. His aunt underwent the procedure 15 years ago.

Yusuf's mom, Yusara Ahmed is pleased with the results. "We were looking forward to this for a long time. It's really amazing."

The medical team sees this treatment as a major advance for medicine Dr. Fish maintains, "We're literally watching medicine change on a day-to-day basis in our lives."

The future is now looking much brighter for this 8-year-old medical pioneer. Yusuf expressed his gratitude, thanking the doctors for taking good care of him.

Doctors feel this is just the beginning. They believe the one-time gene therapy can be used successfully to treat many other diseases, including sickle cell anemia. Ten years in the making, this new treatment marks a major medical milestone.