Children’s Hospital New Orleans Designated for New Sickle Cell Gene Therapy

NEW ORLEANS – From Children’s Hospital New Orleans:

CHNO announced that is 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 Food and Drug Administration (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.

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“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. “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 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 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 CEO of Children’s Hospital. “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.”

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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 Hospital 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 Hospital 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 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. 

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