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SearchThalassemia is a blood disorder that affects the body’s ability to produce healthy red blood cells
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CASGEVY (exagamglogene autotemcel) is a one-time gene therapy for people with sickle cell disease or beta thalassemia.
Cleveland Clinic Children’s is among the first in the country to administer a new gene therapy to modify a patient’s blood-forming stem cells in patients with thalassemia. The one-time treatment can reduce or eliminate the need for ongoing blood transfusions.
The U.S. Food and Drug Administration expanded its approval for Casgevy (exagamglogene autotemcel) in January to treat beta thalassemia, in addition to sickle cell disease. The innovative treatment uses a gene editing technology called CRISPR/Cas9 to modify a patient's own stem cells. The edited cells are then transplanted back into the patient, where they produce more hemoglobin in new healthy red blood cells.
The FDA's approval was based on results from a single arm trial, which showed 91% of patients with beta thalassemia did not need a blood transfusion for at least a year after having the treatment.
“This is the latest game-changing gene therapy in blood disorders that will hopefully greatly improve these patients' quality of life,” said Rabi Hanna, M.D., chairman of the Pediatric Hematology-Oncology department at Cleveland Clinic Children’s. “Oftentimes patients have side effects of their frequent blood transfusions which can lead to organ complications such as liver or heart failure from the severe iron overload. It’s my hope this innovative treatment leads to a significantly better and healthier life for this patient population.”
Thalassemia is an inherited blood disorder that affects the ability to produce normal hemoglobin, which allows red blood cells to transport oxygen throughout the body. Thalassemia can cause extreme fatigue, trouble breathing and dizziness. Standard treatment consists of regular blood transfusions.
Thalassemia affects people who have ancestral links to parts of the world where malaria is prevalent, such as Africa, Southern Europe and West, South and East Asia.
The first patient treated earlier this month was a Northeast Ohio woman, who is doing well. The second was Khalifa Dawood, 22, from Abu Dhabi, who traveled from the United Arab Emirates for the treatment. Khalifa, who looks forward to sharing his story says, “I am hoping this gives me a new lease on life where I am energized to enjoy it.”
Cleveland Clinic Children’s has a specialized center for the care of children with thalassemia and sickle cell. It provides comprehensive care, including treatment and support services., throughout a patient’s lifetime, beginning in childhood.
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