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Warren J. Leonard, MD
Cytokines and Human Immunodeficiency: From Genes to New Therapeutics
Distinguished Investigator, Laboratory of Molecular Immunology, National Heart, Lung, and Blood Institute, National Institutes of Health
2 Riverside Circle, Roanoke, VA 24016
“Cytokines” are hormones that are critical for the normal development and function of cells within the immune system. Cytokines are used clinically, including for expanding T cells for immunotherapeutic use to treat cancer. Defects in cytokines, however, can have profound consequences. Multiple forms of human-inherited immunodeficiency are diseases caused by defective signaling by cytokines. One of these diseases, often known as the “Bubble Boy” disease, results from mutations in the gene that codes for a critical cytokine receptor. Children with the Bubble Boy disease were the first humans to be treated with gene therapy. Dr. Leonard will discuss how discoveries related to this receptor and cytokine signaling led to the identification of other causes of immunodeficiency, as well as the development of a new class of immunosuppressive drugs. He’ll also discuss how ongoing efforts to control cytokine signals has led to the development of next-generation cytokine variants that can inhibit cytokine signaling. The ability to fine-tune cytokine signals allowed Dr. Leonard and his team to find that one such molecule prolongs survival in a model of graft-versus-host disease and inhibits growth of cells from patients with a form of T cell leukemia. The development of such molecules potentially may allow new mechanism-based therapeutic manipulation of the immune system.