Hematology and Oncology | Gene Therapy

“Our oncology program cares for up to 400 new children diagnosed with cancer each year. We provide cutting-edge therapies aimed at improving outcomes and reducing side effects. In fact, our Hematology, Cellular and ImmunoTherapy programs offer curative gene therapy treatments for patients with beta thalassemia and sickle cell anemia. We are among the few qualified treatment centers that provide these FDA-approved ex vivo gene therapy products.”

Andrew Y. Koh, M.D.
Pediatric Hematologist-Oncologist and Division Director of Hematology and Oncology, Children’s Health
Professor, UT Southwestern

PATIENT OUTCOMES

100% 100-day survival rate sibling matched (HLA-identical)
>95% National benchmark

95% 100-day survival rate unrelated allogenic transplants
>95% National benchmark

97.8% Acute lymphocytic leukemia (ALL) survival at five years
>95% National benchmark

KEY PROGRAMS

Gene Therapy

We are among the first centers in the country to offer four gene therapies for sickle cell disease and other rare blood disorders. Before, bone marrow transplant was our only way to help children change the severity of their disorder, and we could only offer it to a fraction of patients.

Provides a certified treatment center for Lyfgenia, Zynteglo, Kymriah and the CAR T-cell therapy for leukemia

Immunotherapy

We’re working to conduct more early-stage trials of immunotherapy-based approaches, with the goal of transforming care for children with some of the most challenging cancers. In fact, we are leading VITAS, one of the first clinical trials in the U.S. to investigate the effect of combining immune checkpoint inhibitors (ICI) with chemotherapy in pediatric patients with solid tumors.

We performed our first gene therapy in 2024 and will be treating 10 patients with gene therapy for a variety of underlying conditions, including sickle cell anemia, beta thalassemia and cerebral adrenoleukodystrophy. We are proud to be one of the highest volume centers for these innovative therapies.