Ellis Neufeld, MD, PhD
|Hospital Title||Associate Chief|
|Academic Title||Professor of Pediatrics|
300 Longwood Avenue
Boston MA 02115
Dr. Neufeld’s main research focus is clinical trials in non-malignant blood disorders, including thalassemia and iron overload, Immune Thrombocytopenia (ITP), and hemophilia. He is presently overall principal investigator for two international phase 2 trials of a novel oral iron chelator.
Dr. Neufeld's laboratory studies candidate genes for inherited blood disorders. His group elucidated the thiamine transporter gene, SLC19A2, as the cause of the syndrome of thiamine-responsive megaloblastic anemia (TRMA) with diabetes and deafness, among other disorders.
About Ellis Neufeld
Ellis Neufeld received his MD and PhD degrees from Washington University in St. Louis. He completed training in Pediatrics, Hematology and Medical Genetics at Boston Children's Hospital and Dana Farber Cancer Institute.
He is presently Associate Chief of the Division of Hematology/Oncology at Children's Hospital, Boston, and Professor of Pediatrics at Harvard Medical School, where he holds the Egan Family Foundation Chair in Transitional Medicine. He is co-chief of the Clinical Research Center at Boston Children’s, and he directs the Boston Hemophilia Center, a joint effort of Children’s and Brigham and Women’s Hospital. He also attends on the nonmalignant hematology service and the thrombosis service and anticoagulation program at Children's Hospital. He has published more than 130 papers, related to his research interests including clinical trials in hemophilia, ITP and thalassemia, and laboratory studies of candidate genes for inherited blood disorders. Dr. Neufeld is the Child Health Program Director for the NIH-sponsored Harvard Catalyst Clinical and Translational Sciences Program.
Bennett CM, Rogers ZR, Kinnamon DD, Bussel JB, Mahoney DH, Abshire TC, Sawaf H, Moore TB, Loh ML, Glader BE, McCarthy MC, Mueller BU, Olson TA, Lorenzana AN, Mentzer WC, Buchanan GR, Feldman HA, Neufeld EJ. Prospective phase 1/2 study of rituximab in childhood and adolescent chronic immune thrombocytopenic purpura. Blood. 2006; 107:2639-42.
Bergmann AK, Sahai I, Falcone JF, Fleming J, Bagg A, Borgna-Pignati C, Casey R, Fabris L, Hexner E, Mathews L, Ribeiro ML, Wierenga KJ, Neufeld EJ. Thiamine-responsive megaloblastic anemia: identification of novel compound heterozygotes and mutation update. J Pediatr. 2009 155:888-892.e1.
Neufeld EJ, Galanello R, Viprakasit V, et al A phase 2 study of the safety, tolerability, and pharmacodynamics of FBS0701, a novel oral iron chelator, in transfusional iron overload. Blood. 2012 119:3263-8.