Gene therapy: A breakthrough for sickle cell anemia?

Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia. The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications.
That's a big change from his situation before the gene therapy, according to his doctors at Necker Children's Hospital in Paris. For years, the boy had been suffering bouts of severe pain, as well as other sickle cell complications that affected his lungs, bones and spleen.
Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia.
It's not clear how long the benefits will last, they said. And the approach obviously has to be tested in more patients.
"This is not right around the corner," said Dr. George Buchanan, a professor emeritus of pediatrics at the University of Texas Southwestern Medical Center in Dallas. That said, Buchanan called the results a "breakthrough" against a disease that can be debilitating and difficult to treat.
Buchanan, who wasn't involved in the research, helped craft the current treatment guidelines for sickle cell. "This is what people have been wanting and waiting for," he said. "So it's exciting."
Sickle cell anemia is an inherited disease that mainly affects people of African, South American or Mediterranean descent. In the United States, about 1 in 365 black children is born with the condition, according to the U.S. National Heart, Lung, and Blood Institute.
It arises when a person inherits two copies of an abnormal hemoglobin gene -- one from each parent. Hemoglobin is an oxygen-carrying protein in the body's red blood cells.
When red blood cells contain "sickle" hemoglobin, they become crescent-shaped, rather than disc-shaped.
 
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Gene therapy: A breakthrough for sickle cell anemia?

Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia. The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications. Thats a big change from his situation before the gene therapy, according to his doctors at Necker Childrens Hospital in Paris. For years, the boy had been suffering bouts of severe pain, as well as other sickle cell complications that affected his lungs, bones and spleen. Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia. Its not clear how long the benefits will last, they said. And the approach obviously has to be tested in more patients. This is not right around the corner, said Dr. George Buchanan, a professor emeritus of pediatrics at the University of Texas Southwestern Medical Center in Dallas. That said, Buchanan called the results a breakthrough against a disease that can be debilitating and difficult to treat. Buchanan, who wasnt involved in the research, helped craft the current treatment guidelines for sickle cell. This is what people have been wanting and waiting for, he said. So its exciting. Sickle cell anemia is an inherited disease that mainly affects people of African, South American or Mediterranean descent. In the United States, about 1 in 365 black children is born with the condition, according to the U.S. National Heart, Lung, and Blood Institute. It arises when a person inherits two copies of an abnormal hemoglobin gene -- one from each parent. Hemoglobin is an oxygen-carrying protein in the bodys red blood cells. When red blood cells contain sickle hemoglobin, they become crescent-shaped, rather than disc-shaped. HealthDay