In a world first, a teenager with sickle cell disease achieved complete remission after an experimental gene therapy at Necker Children's Hospital in Paris, researchers say.
People with sickle-cell disease, a group of inherited blood disorders, have abnormal hemoglobin in their red blood cells, causing blood to clog in the tiny vessels and organs of the body.
After 15 months since treatment, the patient — who began therapy at age 13 — no longer needs medication, and his blood cells show no further sign of the disease, according to a case report published Thursday in The New England Journal of Medicine.
"Since therapy was applied, he hasn't had any pain, any complications. He is free of any transfusions. He plays sports and goes to school," said Dr. Philippe Leboulch, an author of the new research and a professor of medicine at the University of Paris. "So we are quite pleased with the results."