Sickle cell anemia patient ‘cured’ by gene therapy, doctors say

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PARIS – 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.”

This success provides proof of concept for human patients, Leboulch said.

According to Dr. Marina Cavazzana, senior author of the study and head of the biotherapy department at Necker, “all the biological tests we perform lead us to think he is cured.” Yet, she added, the answer to the question of whether he is truly cured “can be provided only by the longer follow-up.”

Still, hopes are running high that patients with this very devastating disease can receive this therapy “in the next five years,” Cavazzana said. “This is our hope, and we work very hard to attain it.”

“Now, we want to be cautious, of course, and we don’t want to say that this is the cure for tomorrow or the next day for everybody,” he said. “At the same time, what we’ve observed is really convincing, and we just hope that we can move this along to make it available to patients,” said Leboulch.