HJNO May/Jun 2024

HEALTHCARE JOURNAL OF NEW ORLEANS  I  MAY / JUN 2024 47 quires a well-matched donor and can be associated with significant complications. A critical advancement for a traditionally underserved population These new gene therapies are a huge leap forward for the children and adults who need treatment most, and lives will be saved and disabilities prevented as we move forward. Historically, research for sickle cell dis- ease has been significantly underfunded compared to diseases that primarily affect White people. for example, cystic fibrosis affects less than a third of the people that sickle cell disease does but receives 27 times the amount of funding per person affected. The FDA approval of the gene thera- pies for sickle cell disease, which have been many years in the making thanks to ground-breaking clinical trials, is an im- portant step in the right direction in ad- dressing health inequities that have been the reality for this underserved population. On the forefront of medicine Only a few centers in the world have administered this ground-breaking ther- apy, and Children’s Hospital New Orleans is currently the only hospital in Louisiana with this distinction for pediatrics and adults and is one of a select number of sites in the country approved for Casgevy. By being an early treatment center that is not available in neighboring states in the Southeast, patients with the most severe forms of sickle cell disease will now have access to the most advanced treatments Benjamin Watkins, MD Director, Pediatric Stem Cell Transplant and Cellular Therapies Program Children’s Hospital New Orleans right here in Louisiana. 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 manufac- turing site where they are used to make the unique gene therapy used to treat the patient. Once ready, the patient receives chemotherapy followed by an IV infusion of the gene therapy. The patient will stay in the hospital for several weeks of recov- ery, to be followed by long-term follow-up care and monitoring. For patients who historically would ex- perience frequent hospital visits, severe pain, and other complications, these trans- formational therapies now provide op- portunity and hope for a more normal life, hopefully with an increased life expectan- cy. There is much to learn as we move for- ward, but we already know through previ- ous clinical trials that the impact of lives will be profound. This provides a gateway for a more normal childhood and healthy adulthood. n BenjaminWatkins,MD, is a nationally recognized pe- diatric hematologist oncologist, who serves as the director of the StemCellTransplant and CellTherapy Programat Children’s Hospital NewOrleans,and as associate professor of Pediatrics atTulane University School of Medicine where he serves as division chief for pediatric hematology oncology.Watkins previous- ly served as associate professor for the Department of Pediatrics,director of the High-Risk Leukemia and Lymphoma Program, and director of the Global On- cology Program at Emory University and the Aflac Cancer and Blood Disorders Center at Children’s Healthcare ofAtlanta.Watkins completed amedical degree and pediatric residency from the University of Arkansas for Medical Sciences (UAMS). He com- pleted a pediatric hematology/oncology fellowship at Emory University School of Medicine and a blood and marrow transplant fellowship at Seattle Chil- dren’s Hospital and the Seattle Cancer CareAlliance. IN DECEMBER, the Food and Drug Administration (FDA) approved two ground-breaking gene therapies, Lyfgenia (bluebird bio) and Casgevy (Vertex), which can now be used in activated centers for treatment of severe forms of sickle cell dis- ease in patients 12 years and older. Children’s Hospital New Orleans is the only authorized treatment center in Loui- siana and among a select group nationally to offer both therapies. Living with sickle cell disease Sickle cell disease is the most common inherited blood disorder affecting 100,000 children and adults in the United States and affects primarily Black or African American people. Eighty babies are born with sickle cell disease in Louisiana every year, and 1,500 children under the age of 18 are living with the disease in the state. Those born with sickle cell disease have a mutation in the gene that makes red blood cells, which are responsible for mov- ing oxygen throughout the body through blood flow. Normal red blood cells are round and travel easily through our blood vessels. In persons with sickle cell disease, red blood cells become sickle shaped and do not travel as well through our blood vessels, often getting stuck together cre- ating blockages. These blockages can lead to severe pain, lung complications, stroke, heart failure, and other life-threatening complications. Because of these ongoing complications and challenges, the life ex- pectancy for a person with sickle cell dis- ease averages around 50 years. Up until now, stem cell transplant has been the only option for cure, but it re-