(COLUMBUS, Ohio) – Nationwide Children’s Hospital delivered its first commercial infusion of CASGEVY™ to 17-year-old Carlos A. from Dayton, Ohio. Today, Carlos is thriving at home, pain-free.
“Carlos has been through a very traumatic journey with his sickle cell disease, so when we presented this gene therapy option to his family, they were excited to pursue it,” said Hemalatha Rangarajan, MD, pediatric stem cell transplant physician at Nationwide Children’s. “The type of gene therapy Carlos has undergone resulted in a reawakening of the fetal hemoglobin, the hemoglobin that is present in utero. Now, Carlos is making his own fetal hemoglobin, which should not cause further sickle cell crisis.”
Carlos was diagnosed with sickle cell disease (SCD) at one year of age. Sickle cell disease is an inherited blood disorder that affects red blood cells, caused by hemoglobin S. Instead of red blood cells that are round, smooth and move easily through the body, carrying oxygen to different parts of the body, patients with SCD have red blood cells that are hard, sticky and sickle-shaped, which can clog blood vessels causing , anemia, pain crisis and damage to the organs from sickle cell crisis that often require frequent hospitalization.
Carlos’ gene therapy journey was one and a half years long, beginning in 2023. The process included multiple consultations with his care teams at both Nationwide Children’s and Dayton Children’s Hospital, monthly exchange transfusions in preparation for his stem cell harvest, two rounds of stem cell mobilization and finally chemotherapy to prepare his body for the gene therapy. The gene therapy infusion itself was complete in less than one hour, and has given Carlos a new sense of hope for his future.
“I want to enjoy life before I get to the grind. I want to go outside for longer, be in extreme temperatures for as long as I want, because my physical strength can be up for it,” said Carlos. “I wish that this would have been available earlier in my life, and I hope that a lot more kids get access to this, not just me.”
Recovery will continue over the coming months, ideally with increased fetal hemoglobin levels and rebounding energy. Over time, Carlos will be able to discontinue some of his medications and return to school.
“Thanks to the active collaboration between Nationwide Children’s and Dayton Children’s, Carlos’ care following discharge will continue closer to his home,” said Mukund Dole, MD, pediatric hematologist-oncologist at Dayton Children’s Hospital and program director of the West Central Ohio Comprehensive Sickle Cell Center in Dayton. “It is truly wonderful to be able to work together in this way to nurture this kind of outcome for a patient with sickle cell disease.”
After being by Carlos’ side through many challenges, his grandmother expressed her joy for this new start.
“We didn’t think people cared enough about sickle cell, because it only affects a certain demographic of Americans. Now, Carlos has his freedom,” said Crystal Sullivan, Carlos’ grandmother. “I just want to do something happy with him. Whatever he wants… we can now do whatever he wants without fear of pain.”
Nationwide Children’s became an authorized treatment center for CASGEVY™ in December 2023. CASGEVY is a CRISPR-based gene therapy that edits a patient’s own blood stem cells to produce high levels of the healthy, oxygen-carrying hemoglobin that is typically produced in utero.
NOTE TO MEDIA: For video and photography assets related to Carlos’ story, visit here.
About Nationwide Children’s Hospital
Named to the Top 10 Honor Roll on U.S. News & World Report’s 2024-25 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 16,000 that provides state-of-the-art wellness, preventive and rehabilitative care and diagnostic treatment during more than 1.8 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.