Clinical trial at The Children’s Hospital at TriStar Centennial paves way for a new sickle cell disease treatment, now approved by FDA

Jan 08, 2024 at 09:17 pm by Staff

NASHVILLE – 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.

About TriStar Centennial Medical Center TriStar Centennial Medical Center is a 741-bed comprehensive facility offering medical and surgical programs including behavioral health, 24-hour emergency, heart and vascular, imaging, neurosciences, oncology, orthopedics, pediatrics, rehabilitation, and women’s services. An affiliate of TriStar Health, TriStar Centennial Medical Center’s 43-acre campus is home to TriStar Centennial Heart & Vascular Center, TriStar Centennial Women’s Hospital, The Children’s Hospital at TriStar Centennial, Sarah Cannon Cancer Institute at TriStar Centennial, TriStar Centennial Advanced Joint Replacement Institute and TriStar Centennial Parthenon Pavilion, one of the largest and most comprehensive private psychiatric facilities in Middle Tennessee. Around the clock care is also available at TriStar Ashland City Medical Center, a rural emergency access hospital in nearby Cheatham County. Opened in 2013, TriStar Spring Hill Emergency Room provides 24-hour emergency care for nearby Spring Hill and surrounding communities.

About TriStar Health

TriStar Health is one of the largest and most comprehensive healthcare providers in the region, comprised of 11 hospitals, 21 imaging centers, 93 physician offices, five freestanding ERs, eight outpatient surgery centers, 21 CareNow® Urgent Care centers and ten medical parks. TriStar Health utilizes advances in clinical technology and innovative procedures while delivering high-quality care. The TriStar Health network had over 900,000 encounters last year, including 478,000 ER visits, 93,000 inpatient admissions and 8,000 babies delivered. The network is supported by over 2,400 physicians and 10,000 employees – including more than 2,800 nurses – who, above all else, are committed to the care and improvement of human life. TriStar Health is an affiliate of HCA Healthcare, one of the nation’s leading providers of healthcare services. Learn more at


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