Margaret MacMillan, MD, MSc

Dr. MacMillan is Professor in Pediatrics in the Division of Blood and Marrow Transplant & Cellular Therapy at the University of Minnesota. Dr. MacMillan received her MSc and MD from the University of Toronto in 1991. She completed residency in Pediatrics and a fellowship in Pediatric Hematology and Oncology at the Hospital for Sick Children at the University of Toronto in 1997. She then completed a fellowship in Pediatric Blood and Marrow Transplantation at the University of Minnesota and joined the faculty in 1999.

Dr. MacMillan’s clinical focus is allogeneic hematopoietic cell transplantation for children with malignant and non-malignant diseases, especially Fanconi anemia. Dr. MacMillan is Director of the Kidz1stFund Comprehensive Fanconi Anemia Center at the University of Minnesota, which funds innovative cellular and gene therapy research for Fanconi anemia. Her research interests also include graft-versus-host disease (GVHD), especially the development and implementation of novel strategies to prevent and treat acute GVHD, and the optimization of acute GVHD clinical trial design by determining GVHD risk and best trial endpoints.

Dr. MacMillan holds 3 INDs for cellular therapy and has an excellent understanding of the regulatory requirements of translational research. She has authored more than 150 peer-reviewed manuscripts and book chapters and is a member of numerous professional societies.

• The ONE Forum 2021 Lives Award, The National Marrow Donor Program (NMDP)/Be The Match (2021)
• Irvine McQuarrie Research Scholar, University of Minnesota (2000)
• Cooley's Anemia Fellowship, Boston, Massachusetts (1997)
• Schilling Scholarship Award in Pediatrics, University of Toronto (1991)
• Charles E. Snelling Scholarship in Pediatrics, University of Toronto (1990)
• Life Sciences Graduate Degree Completion Award, University of Toronto (1988)
• Open Scholarship, University of Toronto (1987)
• Ontario Graduate Scholarship (1986)

1. Acute Graft-Versus-Host Disease
   • T regulatory cells to prevent GVHD
   • Novel pharmacologic agents/ regimens
2. Fanconi anemia
   • Novel preparative therapies
   • Gene therapy - multipotent adult stem cell
   • Mechanisms to speed immune recovery and reduce infection after transplantation
3. Juvenile Myelomonocytic Leukemia
   • Novel phase I agents
   • Novel preparative therapies