Physicians and researchers at Cincinnati Children’s conduct numerous clinical trials in hopes of improving the lives of patients with sickle cell disease and other hemoglobin disorders. Patients have the opportunity to participate in these studies, as well as in those offered at our site through organizations such as the National Heart, Lung and Blood Institute.

Outstanding laboratory research at Cincinnati Children’s provides the foundation for innovative clinical trials. Researchers at Cincinnati Children’s are exploring a variety of lab research initiatives.

  • Punam Malik, MD, the center director, is known nationally, and even internationally, for her gene therapy research. She also is the Program Leader of the Hematology and Gene Therapy Program at the Cancer and Blood Diseases Institute.
  • Theodosia Kalfa, MD, PhD, studies the structure of red blood cell membranes, how they are affected by sickle cells, and ways to improve membrane function in a variety of blood diseases.
  • Ajay Perumbeti, MD, the current Sickle Cell Research Scholar, is working with Dr. Malik to develop gene therapy as a cure for sickle cell disease. They are exploring ways to remove a sickle cell patient’s blood-producing cells from the bone marrow, correct the sickle gene, and return the cells back into the bone marrow to stimulate production of normal hemoglobin

Sickle Cell Research Scholar

The Comprehensive Sickle Cell Center receives funding from the National Institutes of Health to support a young faculty member who is focusing his or her research career on sickle cell disease.  There are only 11 such programs across the country.

The current Sickle Cell Research Scholar is Ajay Perumbeti, MD, whose work focuses on gene therapy for sickle cell disease.