Researchers have reported an early victory in treating sickle cell anemia with a new gene therapy. This could possibly lead to a cure.
According to the U.S. National Heart, Lung, and Blood Institute, one in 365 black babies are born with the disease. This is an inherited disease that affects people of African, Mediterranean, and South American decent.
A teenager in France received the gene therapy. After 15 months, he no longer had any symptoms associated with the condition. His doctors at a children’s hospital reported that they saw a big change in his condition after having the gene therapy. Before, he suffered from rounds of severe pain. And because of the disease, he had complications in other parts of his body, such as the bones, spleen, and lungs.
With gene therapy, the purpose is to alter a patient’s blood stem cells genetically so they do not make abnormal hemoglobin. In this case, the teenager’s doctors were focused on beta globin, which is mutated in people with sickle cell anemia.
Although the gene therapy was effective on one patient, it needs to be tested on other patients. There needs to be more research before this can becomes a standard option for others with sickle cell anemia. These results are considered a “breakthrough” for a disease that is devastating and hard to treat.
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