Margaret MacMillan

Professor, Division Director, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy
Faculty, Department of Pediatrics
BMT Specialist, Pediatric Blood and Marrow Transplant (BMT) Center
Vice-President, Board of Directors of the American Society for Transplantation & Cellular Therapy


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.

Research Summary

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

Clinical Summary

Clinical specialties: Blood and Marrow Transplantation, Fanconi Anemia, Juvenile Myelomonocytic Leukemia


MD, University of Toronto Medical School, Ontario, Canada
MSc in Nutritional Sciences, University of Toronto, Ontario, Canada

Fellowships, Residencies, and Visiting Engagements

Fellowship in Hematology/Oncology,
The Hospital for Sick Children
Toronto, Ontario, Canada
Residency in Pediatrics,
The Hospital for Sick Children
Toronto, Ontario, Canada
Fellowship in Blood and Marrow Transplantation,
University of Minnesota Medical School
Minneapolis, Minnesota, United States

Licensures and Certifications

Pediatrics, American Board of Pediatrics

Honors and Recognition

Charles E. Snelling Scholarship in Pediatrics, University of Toronto
Schilling Scholarship Award in Pediatrics, University of Toronto
Irvine McQuarrie Research Scholar, University of Minnesota
Cooley's Anemia Fellowship, Boston, MA
The ONE Forum 2021 Lives Award, The National Marrow Donor Program (NMDP)/Be The Match



Pediatric Blood and Marrow Transplantation & Cellular Therapy
Mayo Mail Code 366
420 Delaware Street SE
Minneapolis, MN 55455

Administrative Contact

Joyce Selle
Administrative Phone: 612-625-7117
Administrative Email:
Administrative Fax Number: 612-626-4074