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The Children’s Hospital at TriStar Centennial paves way for a new sickle cell disease treatment

Physicians and researchers at The Children's Hospital at TriStar Centennial and Sarah Cannon Research Institute (SCRI), the research partner of HCA Healthcare, led a clinical trial for CRISPR-Cas9 gene-editing therapy to treat sickle cell disease.

January 09, 2024

Nashville, Tenn. — Physicians and researchers at The Children’s Hospital at TriStar Centennial and Sarah Cannon Research Institute (SCRI), the research partner of HCA Healthcare, led a clinical trial for CRISPR-Cas9 gene-editing therapy to treat sickle cell disease. Vertex Pharmaceuticals, who developed the therapy with CRISPR Therapeutics, recently received approval from the Food and Drug Administration (FDA) to treat sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs), making it the first CRISPR gene-editing therapy approved to treat any disease.

Led by Dr. Haydar Frangoul, medical director of pediatric hematology/oncology at SCRI and the Sarah Cannon Pediatric Hematology/Oncology & Cellular Therapy Program at HCA Healthcare’s The Children’s Hospital at TriStar Centennial, the hospital began treating the first patient with sickle cell disease using CRISPR gene-editing therapy in 2019. The Children’s Hospital at TriStar Centennial was a leader among clinical trial sites for sickle cell disease patients.

“This therapy can truly make a difference for patients with sickle cell disease,” said Dr. Frangoul. “Patients I have treated can now live without acute pain or visits to the hospital.”

Sickle cell disease is an inherited blood disorder that affects approximately 100,000 individuals in the United States. Sickle cell disease is debilitating and results in recurrent pain episodes, organ damage such as stroke and kidney failure, and shortened life expectancy.

CRISPR therapy involves collecting a patient’s stem cells and gene editing them to produce high levels of fetal hemoglobin, which reduces the production of abnormal red cells typically seen in patients with sickle cell disease and prevents occurrence of debilitating and life-threatening manifestations.

“Our team helped pioneer this therapy to help individuals with sickle cell disease who are suffering on a daily basis. With the FDA approval, we can begin treating patients within The Children’s Hospital at TriStar Centennial in the near future,” said Dr. Frangoul.

For more information about the therapy, please call (615) 342-7339.

Published:
January 09, 2024
Location:
The Children's Hospital at TriStar Centennial