Gene therapy designed to fight sickle cell disease is effective in two patients. It was presented at the annual meeting of the American Society of Hematology (ASH 2018).
After more than a decade of research, a gene therapy designed to fight sickle cell disease is clearing symptoms of the disease in two people with the disease.
Sickle cell disease is a genetic disease that affects the hemoglobin of red blood cells. Very widespread, it is manifested in particular by anemia (jaundice, permanent fatigue), very violent pain flare-ups in the joints and an increased risk of infections. Many die from it. Current treatments have greatly increased the life expectancy of affected patients, but they remain limited.
Elimination of chronic pain
“One year after treating our first patient, and six months after treating our second patient, both have seen a remarkable improvement in their quality of life with a significant reduction in disease symptoms. This includes near- elimination of chronic pain and improvement of anemia,” said Malik Punam, director of the study and the Comprehensive Sickle Cell Center. “These preliminary results are very promising for all patients with sickle cell disease,” she adds.
The two patients in question are 35 and 25 years old. A year after the treatment, they no longer need opioids to calm their pain, whereas before they suffered about one flare-up per month.
Fetal hemoglobin
Gene therapy involves introducing genetic material into cells to cure a disease. Here, the team placed a gene for fetal hemoglobin in the bone marrow cells of patients. This allows the body to produce normal red blood cells instead of the sickle-shaped cells responsible for hereditary pathology.
Sickle cell disease particularly affects populations from sub-Saharan Africa, the West Indies, North America, the Mediterranean basin, the Middle East and India.
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