Sickle Cell Disease

Pursuing New Therapies Through Research

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.

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.

Clinical Trials Opportunities

Physicians and researchers at Cincinnati Children’s offer clinical research studies for patients with sickle cell disease and other hemoglobin disorders.

We are currently building a blood and tissue bank so that we can develop better treatments for sickle cell disease. We are also testing the drug Zileuton to see if it can reduce the number of acute sickle events in patients with sickle cell disease.

Featured Clinical Trials for Sickle Cell and Hemoglobin 


Clinical Trials.

Find a clinical study specific to sickle cell disease and hemoglobin disorders.

More About Our Research

Learn more about our world-class research on cancer in the Division of Hematology. Read More