(COLUMBUS, Ohio) – Nationwide Children’s Hospital has been designated a qualified treatment center for Zynteglo® (betibeglogene autotemcel), a gene therapy approved for the treatment of people with beta thalassemia who require regular red blood cell transfusions.
In August 2022, the U.S. Food and Drug Administration approved Zynteglo, which was developed by gene therapy company bluebird bio, Inc., for the treatment of beta thalassemia in adult and pediatric patients who require regular red blood cell tranfusions.
As a designated treatment center Nationwide Children’s becomes part of a network of highly specialized facilities throughout the U.S. that is certified to have the advanced facilities and specially-trained staff necessary for the administration of Zynteglo.
“This one-time treatment can change the lives of patients who suffer from transfusion dependant beta thalassemia,” said Hemalatha Rangarajan, MD, pediatric stem cell transplant physician at Nationwide Children’s. “To have a new treatment option for families that offers the possibility of stopping a cumbersome monthly transfusion schedule provides families hope for a new sense of normal.”
Beta thalassemia is an inherited blood disorder that limits the body’s ability to produce beta-globin, a protein needed to make hemoglobin and red blood cells. Patients with transfusion-dependent beta thalassemia receive monthly blood transfusions as part of their treatment, in order to supply red blood cells needed to carry oxygen throughout the body.
The gene therapy is a type of therapy called gene addition. Each dose of Zynteglo is a customized treatment that uses a patient’s own cells that are modified to produce functional beta-globin. In two phase 3 open-label studies, 41 patients received Zynteglo. About 89% of study participants treated were able to stop transfusions.
“This is the kind of therapy we are always striving to provide, care that is personalized and specific,” said Rolla Abu-Arja, MD, director of the Blood and Marrow Transplant (BMT) program at Nationwide Children’s. “Because this gene therapy is created using a patient’s own cells, we are thrilled to be able to ensure such individualized care.”
About Nationwide Children’s Hospital
Named to the Top 10 Honor Roll on U.S. News & World Report’s 2022-23 list of “Best Children’s Hospitals,” Nationwide Children’s Hospital is one of America’s largest not-for-profit free-standing pediatric health care systems providing unique expertise in pediatric population health, behavioral health, genomics and health equity as the next frontiers in pediatric medicine, leading to best outcomes for the health of the whole child. Integrated clinical and research programs, as well as prioritizing quality and safety, are part of what allows Nationwide Children’s to advance its unique model of care. Nationwide Children’s has a staff of more than 13,000 that provides state-of-the-art wellness, preventive and rehabilitative care and diagnostic treatment during more than 1.6 million patient visits annually. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s physicians train the next generation of pediatricians and pediatric specialists. The Abigail Wexner Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded free-standing pediatric research facilities. More information is available at NationwideChildrens.org.