Children’s Hospital New Orleans designated as Louisiana’s only treatment center for newly approved sickle cell disease gene therapy
The hospital is the pediatric facility in the Gulf South to offer this lifesaving therapy
NEW ORLEANS – Children’s Hospital New Orleans is proud to be the only hospital in Louisiana to serve as a designated treatment center for both Lyfgenia (BlueBird Bio) and Casgevy (Vertex), two gene therapies whose products were recently approved by the FDA to treat Sickle Cell Disease in patients 12 years and older.
This ground-breaking gene therapy is now available for patients with Sickle Cell Disease, an inherited blood disorder affecting approximately 80 babies born in Louisiana every year. The Louisiana Department of Public Health estimates that there are at least 3,000 people with Sickle Cell Disease living in the state. Sickle Cell Disease affects the red blood cells primarily of people of African, Latin American, and Mediterranean heritage. It poses a risk for serious lifelong medical complications and shortened life expectancy.
“Our Stem Cell Transplant and Sickle Cell teams have worked incredibly hard for decades to provide the foundational excellence that makes these therapies possible for the patients and families we serve,” said Dr. Charles Hemenway, Service Line Chief for the Center for Cancer and Blood Disorders at Children’s Hospital New Orleans. “These gene therapies are a leap forward for the children who need treatment most, and lives will be saved and disabilities prevented as we move forward.”
As part of its initial testing, approximately 100 patients in the world have received this ground-breaking therapy. Children’s Hospital New Orleans is currently the only hospital in Louisiana that has been approved by the manufacturers to treat patients with their life-changing gene therapy products. Children’s Hospital New Orleans is one of only 13 sites in the country approved for Casgevy and is also now one of the only hospitals in the Southeast and the only hospital in Louisiana to be an authorized center for Zynteglo (Bluebird Bio), an FDA-approved gene therapy for the treatment of beta-thalassemia.
“This is an important step in the right direction in addressing health inequities that have been the reality for this underserved population,” said Lucio A. Fragoso, President and Chief Executive Officer of Children’s Hospital New Orleans. “By being an early treatment center that is not available in neighboring states in the Southeast, our patients with the most severe presentations of Sickle Cell Disease will now have access to the most advanced treatments right here at home. This is an amazing opportunity afforded to the children of Louisiana and the Gulf South region.”
These gene therapies are made using a patient’s own blood stem cells. The stem cells are collected using a process called apheresis, and then sent to a manufacturing site where the genetic makeup of the cells is altered such that they produce healthy red blood cells. Once ready, the patient receives an IV infusion of the “engineered” stem cells and is monitored in the hospital for several weeks awaiting growth of the transplanted cells. This is followed by long term follow-up care and monitoring.
The Center for Cancer and Blood Disorders at Children’s Hospital provides advanced treatments for children with leukemia, lymphoma, sickle cell anemia, hemophilia, and other childhood cancers and blood disorders. Children’s is the only accredited pediatric stem cell transplant facility in the state, performing bone marrow transplants and CAR-T cell therapy, in addition to the newly approved gene therapies for Sickle Cell Disease. Children's is also actively conducting cancer research at the forefront of medicine and has the largest, most experienced group of pediatric cancer specialists in the region, staffed by faculty from LSU Health New Orleans and Tulane University School of Medicine.
Children’s Hospital New Orleans cared for 20 transplant patients in 2023 and expects this population to grow as the hospital continued to develop its Stem Cell Transplant and Cellular Therapy Program. Learn more at www.chnola.org.
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About Children’s Hospital New Orleans:
Children’s Hospital New Orleans is a 263-bed, non-profit academic pediatric medical center that offers comprehensive healthcare services, including over 40 pediatric specialties, delivered just for children. With more than 600 pediatric providers, Children’s offers a comprehensive array of pediatric healthcare services in Louisiana and the Gulf South. In addition to its main campus located in New Orleans, the hospital operates a network of specialty clinics across Louisiana, including in Covington, Baton Rouge, Alexandria, Lafayette, and Diamondhead, MS. Children’s offers primary care services at 14 convenient locations and via its Virtual Care for Kids program. Children’s is a proud member of LCMC Health, a Louisiana-based, not-for-profit hospital system which also includes New Orleans East Hospital, Touro, University Medical Center New Orleans, West Jefferson Medical Center, East Jefferson General Hospital, Tulane Medical Center, Lakeview Hospital, and Lakeside Hospital. Learn more at chnola.org.