Earlier this summer, AAMDSIF called for research proposals addressing Paroxysmal Nocturnal Hemoglobinuria (PNH).
Robert A. Brodsky, MD, is the Johns Hopkins Family Professor of Medicine and Oncology and Director of the Division of Adult Hematology. He has a long track record of funded research through the NIH and through foundation grants, and serves as the Director of the Johns Hopkins Hematology Fellowship program, as well as the PI for the Johns Hopkins T32 in hematology and co-PI of a K12 in benign hematology. His focus on PNH and interest in translational medicine led his laboratory to develop a novel diagnostic reagent (FLAER) used in PNH.
His research project that was awarded funding in 2015 is titled Preclinical Development of an Oral Factor D Complement Inhibitor to Treat PNH. PNH is a life-threatening hemolytic anemia that results from the loss of cell surface complement regulators. The monoclonal antibody eculizimab was developed to combat the thrombosis that constitutes a leading cause of death in PNH, but produces a suboptimal response in approximately 20% of the population, including the 1-2% of patients of Asian origin who are resistant to the drug. In addition, eculizimab is an intravenous drug that is administered every 14 days. In his work, Dr. Brodsky proposes to develop a novel complement inhibitor that could form the basis of an oral drug to block the continued hemolysis common among PNH patients and eliminate the need for frequent intravenous dosing with current regimens.
Through the generous support of the Geczik family, this award provides funding for one year to support the development of this new drug in the treatment of PNH, which if successful could significantly reduce the cost of treatment and improve daily quality of life for patients.