HCA Healthcare Announces Promising Gene-Editing Therapy for Pediatric Blood Disorders
TL/DR –
HCA Healthcare has announced research showing promising results for a gene-editing therapy in children aged 5-11 with severe sickle cell disease and transfusion-dependent beta thalassemia. The study evaluated exa-cel, a CRISPR-based cell therapy which edits a patient’s blood-forming stem cells to increase the production of fetal hemoglobin, helping prevent or reduce disease complications. The findings were based on two phase 3 studies that enrolled 26 children; all children with beta thalassemia achieved transfusion independence for at least 12 months, while all children with sickle cell disease remained free from severe vaso-occlusive crises for at least 12 months.
HCA Healthcare Unveils Promising Gene-Editing Therapy Results for Children with Sickle Cell Disease and Beta Thalassemia
HCA Healthcare has released research in The New England Journal of Medicine, showcasing promising outcomes of a gene-editing therapy for children aged 5-11 with severe sickle cell disease and transfusion-dependent beta thalassemia. The treatment for these inherited blood disorders, prevalent in early childhood, underscore the merits of gene-editing therapy and the necessity for continuous clinical research.
A Collaborative Effort
Dr. Haydar Frangoul, medical director of HCA Healthcare’s Sarah Cannon Transplant and Cellular Therapy Program at TriStar Centennial Children’s Hospital, spearheaded the research in partnership with Sarah Cannon Research Institute. The team evaluated the efficacy of exagamglogene autotemcel (exa-cel) in young patients.
Addressing Prevalent Blood Disorders
Sickle cell disease, the most common inherited blood disorder in the U.S., affects nearly 100,000 people, causing severe pain, organ damage, and frequent hospitalizations in children. Similarly, severe beta thalassemia affects thousands of children, leading to significant health issues due to lifelong blood transfusions.
About the Gene-Editing Therapy
The FDA-approved exa-cel therapy uses CRISPR technology to edit a patient’s blood-forming stem cells, increasing production of fetal hemoglobin to combat disease complications. The study included 26 children, all of whom achieved transfusion independence and remained free from severe vaso-occlusive crises for at least 12 months.
HCA Healthcare’s Role in Advancing Gene Therapy
The study exemplifies HCA Healthcare’s pioneering efforts in cell and gene therapy. Dr. Frangoul’s previous contributions to the first U.S. clinical trial to use gene-editing for treating sickle cell disease helped develop the first FDA-approved CRISPR-based therapy for patients over 12 years.
Dr. Michael Cuffe, executive VP and chief clinical officer of HCA Healthcare, emphasized the importance of such research in advancing medicine and treatment possibilities.
Expanding Access to Gene-Editing Therapies
Building on this research, HCA Healthcare’s Sarah Cannon Transplant and Cellular Therapy Network is broadening access to FDA-approved gene-editing therapies. TriStar Centennial Children’s Hospital and Methodist Children’s Hospital currently offer these therapies, with Medical City Children’s Hospital in Dallas preparing to expand services.
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