Clinical trial at Levine Children’s Hospital studies Gene Therapy as possible cure for Sickle Cell Disease

Sickle Cell Awareness Month

CHARLOTTE, N.C. (WBTV) - Atrium Health’s Levine Children’s Hospital is opening a new clinical trial to cure Sickle Cell Disease.

Pediatric Hematologist and Oncologist Dr. Kristina Harrell says Sickle Cell Disease is the most common, inherited, blood disorder.

Patients with Sickle Cell Disease have banana or crescent-shaped blood cells, instead of donut-shaped blood cells. The abnormal cells can cause blockages, pain, inflammation, and damage to a person’s internal organs.

Bone Marrow Transplants are the only cure for Sickle Cell Disease. Doctors perform Bone Marrow Transplants at Levine Children’s Hospital, but Dr. Harrell says it is a complex and invasive procedure, that relies on a donor match to be successful.

Dr. Harrell says they are opening a clinical trial with the hopes of finding another cure for Sickle Cell Disease. Levine Children’s Hospital will soon begin researching Gene Therapy. Dr. Harrell says this procedure is much less invasive. Doctors will extract a patient’s stem cells, alter them to remove the Sickle Cell Disease, then infuse the stem cells back into the patient.

“If we change those stem cells so that they don’t have the sickle cell defect anymore, they put another change in that makes the sickle cell disease less complicated or severe, that will either cure children and young adults or it will cause their sickle cell to not cause many complications,” Dr. Harrell explained.

Dr. Harrell says this clinical trial is one of many happening in the U.S. She says they are in the early stages of the trial.

For patients who do not receive a Bone Marrow Transplant to cure Sickle Cell Disease, they must make frequent visits to the hospital in order to live with Sickle Cell Disease. Dr. Harrell says it isn’t always easy to make frequent trips to Levine Children’s Hospital because some patients live nearly three hours away.

Dr. Harrell says Atrium Health will be using grant funding from Global Blood Therapeutics to educate primary care providers on Sickle Cell Disease. Patients can visit their primary care providers in between trips to Levine Children’s Hospital to reduce the frequent travel.

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