Paris – Doctors at the Necker Children’s Hospital revealed that the sickle cell anemia is curable with gene therapy. The disclosure was done after a boy afflicted with sickle cell disease attained complete cure after a genetic engineering therapy treatment.
The sickle cell anemia is a blood disorder which principal sign is an abnormal hemoglobin count in the red blood cells, causing blood to clot in the vessels and organs of the body. Those patients have a shortened life span, reduced by decades compared to the general population, according to the American Sickle Cell Anemia Association.
This patient, who began therapy at 13 years old, no longer needs medication, and his blood cells show no sign of the disease, after 15 months of treatment. The information was published by The New England Journal of Medicine.
“Since therapy was applied, he hasn’t had any pain, any complications (…) He plays sports and goes to school”, said Dr. Philippe Lebouch, professor of medicine at the University of Paris and one of the authors of this new research to CNN. “So we are quite pleased with the results,” he said.
Dr. Marina Cavazanna, senior author of the study and head of the biotherapy department at Necker Children’s Hospital, said that “all biological tests we perform lead us to think he is cured,” according to The Chicago Tribune. However, she added that the answer to the question of whether he is cured could be provided only by further follow-ups.
Sickle cell disease is one of the most common disorders in the world, a genetic mutation causes hemoglobin to distort the shape of the cell, and this causes the blood to aggregate or clog. “So what we did here was, we tried to inhibit the process of aggregation.” Dr. Leboulch said.
This treatment consists in extracting bone marrow from the patient, harvest the stem cells and alter the genetic instructions so that they would produce normal hemoglobin. Next, the researchers treated the patient with chemotherapy for four days to eliminate his diseased stem cells, and finally, they returned the treated stem cells into patient’s bloodstream.
Worldwide, more than 275,000 infants are born with sickle cell disease each year. In the United States, approximately 100,000 people, most of Afro-American or identifying as black, currently have it. About one in every 365 black children in the US is born with sickle cell disease, for which life expectancy is now about 40 to 60 years.
“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,” Leboulch 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,” He added.