“Since we see kids from birth, we develop relationships with the family, and the family gets to know us,” Dr. Quinn said. “We’re always available. They know that they can call us, and we can mobilize the services and the resources that we have for them.”
Advancing Care—and a Cure—for Sickle Cell Disease
Sickle cell research at Cincinnati Children’s has its roots in the center’s early days, when leading physicians advocated for the use of penicillin prophylaxis which greatly improved the survival of patients, including Denae.
More recently, Cincinnati Children’s researchers developed gene therapy for sickle cell disease, a novel cure that harvests stem cells from the patient's bone marrow and genetically engineers them to produce fetal hemoglobin. While bone marrow transplant has been an option to cure sickle cell disease, it is only available to a small number of children who have a matched donor.
“Every patient is their own donor because we fix the patient’s own bone marrow stem cells,” Dr. Malik said. “That means every patient with sickle cell has this option.”
This treatment could be administered once and last a lifetime, eliminating the need for daily hydroxyurea. Two of these genetic therapies have now received U.S. Food and Drug Administration (FDA) approval for the treatment of sickle cell disease.
This research ultimately comes to the bedside for the benefit of patients like Denae and, decades later, Mariam, empowering them to live pain-free.
“I hope the best thing that we’ve been able to give to Mariam is a normal childhood,” Dr. Quinn said. “She hasn’t had severe pain or complications from her sickle cell disease. She’s going to school. She’s growing and developing well. It’s the things that she’s avoided that are helping her stay healthy.”
For Denae, it comes back to the support she and her family have received for more than 30 years.
“When I think about why we are at Cincinnati Children’s it’s because I think is the best place for her to be,” Denae said. “I know that they’re just a call away. I’m going to speak to someone that day, and I think that that level of care isn’t always common, but it’s common for us. People travel all over just to come to a hospital that we literally have right in our neighborhood.”
For Mariam, the benefit is much simpler: “They made me feel better. And I get to go home.”
(Published March 2025)