Margaret MacMillan, MD, MSc

Professor, Department of Pediatrics

Margaret MacMillan

Contact Info

Office Phone 612-626-2961

Fax 612-626-2815

Office Address:
Pediatric BMT
Mayo A537
420 Delaware St SE
Minneapolis, MN 55455

Mailing Address:
Pediatric BMT
MMC 484
420 Delaware St SE
Minneapolis, MN 55455

Administrative Assistant Name
Joyce Selle

Administrative Phone

Administrative Email

Administrative Fax Number

MD, University of Toronto Medical School, Ontario, Canada

Residency in Pediatrics, The Hospital for Sick Children, University of Toronto, Ontario, Canada

Fellowship in Hematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada

Fellowship in Blood and Marrow Transplantation, University of Minnesota Medical School, Minneapolis, MN

MS, Nutritional Sciences, University of Toronto, Ontario, Canada


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.

Awards & Recognition

  • 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)


Research Summary/Interests

  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



  • Blood and marrow transplantation, Fanconi&rsquo
  • s anemia, Juvenile myelomonocytic leukemia

Board Certifications