HJNO Nov/Dec 2025

HEALTHCARE JOURNAL OF NEW ORLEANS  I  NOV / DEC 2025 43 Ben Watkins, MD Director of Pediatric Stem Cell Transplant & Cellular Therapies Manning Family Children’s tember. This marks the first time the pro- cess of using gene therapy for treatment of sickle cell disease was begun in Louisiana or the Gulf South on a pediatric or adult patient — a historical moment for our state, and most importantly, those whose lives will be significantly changed. Daniel’s stem cells are currently going through the process of being edited at the laboratory overseas, and when ready, will be sent back for his transplant process to begin early next year. The edited cells will then be reintroduced into his body, with the goal of allowing him to produce healthy, normally shaped red blood cells. A Chance to Fly For Daniel, this is not just a chance to be cured of this debilitating disease; it is also an opportunity to achieve his dream of be- coming a commercial airline pilot. He has completed all of his training, but has been told he can only obtain a pilot’s license if he is cured of his disease through gene therapy. Once the transplant of his edited genes takes place, his dream of flying will final- ly be within reach. And then, the sky’s the limit. n Benjamin Watkins, MD, is a pediatric hematologist oncologist, serving as the director of the stem cell transplant and cell therapy program at Manning Family Children’s. He is also an associate professor of pediatrics atTulane University School of Medicine, where he serves as the division chief for pediatric hematology oncology. IN early 2024, Manning Family Children’s became the first hospital in Louisiana and the Gulf South to serve as an authorized treatment center for both Lyfgenia and Casgevy, two gene therapies approved by the FDA in late 2023 to treat sickle cell dis- ease in patients age 12 and older. Sickle cell disease, an inherited blood disorder affecting over 100,000 children and adults each year in the U.S., affects the red blood cells primarily of people of Afri- can descent and Latin American and Med- iterranean heritage. It puts those with the disease at risk for lifelong complications and shortened life expectancy. One out of 365 African American newborns is affect- ed. Here in Louisiana, there are more than 3,000 people living with sickle cell disease, one of the highest per capita in the United States. Manning Family Children’s follows approximately 500 children and young adults with sickle cell disease, the most in the Gulf South. Medical History in the Making These gene therapies are made using a patient’s own blood stem cells. The pa- tient’s stem cells are collected using a pro- cess called apheresis, and then sent to a manufacturing site where they are used to make the unique gene therapy to treat that patient. Once ready, the patient receives an IV infusion of the gene therapy and stays in the hospital for several weeks of recovery. After discharge, the patient receives long- term follow-up care and monitoring. Louisiana’s first patient to undergo this gene therapy is 22-year-old Daniel Cressy, who has been followed by the team at Manning Family Children’s since birth. He was started on the standard of care management for his sickle cell disease (hydroxyurea) as a young child and has re- mained on it since. Despite compliance with his medica- tion, he has suffered from severe chronic pain and has required ER visits and inter- mittent admission to the hospital for his pain. Daniel’s pain significantly worsened in his young adulthood, and in the past two years he has had many visits to the ER and several prolonged hospitalizations for se- vere pain. The complications of his sickle cell dis- ease have not only disrupted his life, they have prevented him from achieving his dreams. Daniel was initially evaluated for a stem cell transplant, but he had no sibling or donor matches. His only hope was for gene therapy. The hospital began the lengthy insur- ance authorization process for Daniel’s gene therapy in fall of 2024 and autho- rizations, including arrangements for purchasing of the drug Casgevy, were ap- proved through a specialty pharmacy in April of this year, allowing the process to move forward. The first collection of his stem cells took place in late July 2025, followed by a sec- ond round of stem cell collection in Sep-