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Walters, Mark C., MD

Mark Walters

MD

Pediatric Hematology-Oncology

Program Director, Blood & Marrow Transplantation (BMT)

Program Director, Cord Blood Program

Medical Director, Jordan Family Center for BMT & Cellular Therapies Research

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Clinical Specialties

Cancer, tumors, sarcoma, chemotherapy, targeted therapy, brain and spinal cord tumors, pediatric oncology, hepatoblastoma, wilms tumor blood and bone marrow cancers, blood disorders, anemia, clotting disorders, sickle cell disease, hemophilia, leukemia and lymphoma, hodgkins and non-hodgkins, stem cell transplantation

Certifications

Board Certification
  • Hematology/Oncology, Pediatrics

Education

Medical School

University of California, San Diego School of Medicine, CA

Fellowship

University of Washington Medical Center, WA
(Pediatric Hematology/Oncology)

Fellowship

Fred Hutchinson Cancer Research Center, WA
(Pediatric Hematology/Oncology)

Residency

University of Washington Medical Center, WA
(Pediatrics)

Internship

University of Washington Medical Center, WA

Clinic Locations

My Work

About Me

Dr. Walters is the Jordan Family Director of Bone Marrow Transplantation at UCSF Benioff Children’s Hospital Oakland. He has been the Director of Stem Cell Therapy and a Division leader since 1999. He was recruited from the University of Washington and Fred Hutchinson Cancer Research Center. With R01 NIH funding, he initially focused on laboratory research in the area of transcriptional gene expression. During this period, he expanded his responsibilities to develop a career in stem cell transplantation in sickle cell disease. The integration of these areas of expertise has enabled him to develop a national program focusing on novel therapies for blood disorders.

Since joining Children’s Hospital, he has devoted his research career to pursuing curative therapies for hemoglobin disorders with an overarching goal of expanding this treatment more broadly to affected individuals. With NIH and industry support, he has conducted a number of multicenter clinical trials for sickle cell disease and thalassemia. Currently, he is focused on initiating early phase clinical trials to test novel genomic editing techniques in hematopoietic stem cells (HCT) from individuals affected by hemoglobin disorders, starting with a high-profile trial in sickle cell disease. Other projects in the pipeline include activation of fetal hemoglobin by genomic editing in stem cells that might be applied more universally to all hemoglobin disorders regardless of genotype and in utero transplantation for alpha thalassemia major.

Dr. Walters is co-PI of two BMT – CTN trials, one funded under a MPI U01 mechanism through NHLBI (STRIDE). These studies aim to broaden the application of conventional HCT for hemoglobin disorders by comparing HLA-identical and well-matched URD bone marrow transplant to a cohort of non-transplant subjects who lack a donor and to test HLA-haploidentical HCT in children and adults with severe sickle cell disease. Dr. Walters has worked with industry sponsors and taken a lead investigator role in an early phase clinical trials of gene therapy for thalassemia major and SCD (Bluebird Bio, Inc). He is also the clinical lead investigator of a planned trial to inactivate bcl11a in hematopoietic stem cells to re-activate fetal hemoglobin expression in subjects with thalassemia major (Sangamo, Biogen).

Dr. Walters is a national scientific, clinical and educational leader in hematology. He has been the principal investigator of the NHLBI Sickle Cell Disease Clinical Research Network, Chair of the Non-malignant Disease Strategy Group for the Pediatric Bone Marrow Transplant Consortium (PBMTC), and an active member of NIH study sections. Presently he is the Chair of the NHLBI Sickle Cell Disease Advisory Committee. Regionally, he is leading many activities in the UC Berkeley/Children’s Hospital CIRM stem cell and training program. Dr. Walters is an exceptional teacher and has mentored many post-doctoral candidates and fellows.

Professional Achievements
  • National and international speaker for hematopoietic (blood) cell transplantation
  • American Society of Hematology, Scholar Award/Jose Carreras Award
  • NIH Clinical Investigators Development Award
  • Principal Investigator, Sickle Cell Disease Clinical Research Network
  • Co-Chair, Non-malignant diseases Working Committee, CIBMTR
  • Vice-Chair, Non-malignant Disorders Committee, PBMTC

Professional Organizations

  • Member, American Society of Hematology
  • Member, American Society of Blood and Marrow Transplantation

Selected Research and Publications:

  1. Walters MC, De Castro LM, Sullivan KM, Krishnamurti L, Kamani N, Bredeson C, Neuberg D, Hassell KL, Farnia S, Campbell A, Petersdorf E. Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease. Biol Blood Marrow Transplant 2016;22 2:207-11.
  2. Walters MC, MacKenzie TC. A booster shot to cure hemoglobinopathies. Blood 2015;126 10:1159-61.
  3. Walters MC. Update of hematopoietic cell transplantation for sickle cell disease. Curr Opin Hematol 2015.
  4. Derderian SC, Jeanty C, Walters MC, Vichinsky E, MacKenzie TC. In utero hematopoietic cell transplantation for hemoglobinopathies. Front Pharmacol 2015;5:278.
  5. Walters MC, De Castro LM, Sullivan KM, Krishnamurti L, Kamani N, Bredeson C, Neuberg D, Hassell KL, Farnia S, Campbell A, Petersdorf E. Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease. Biol Blood Marrow Transplant 2015.
  6. Walters MC, Rasko J, Hongeng S, Kwiatkowski J, Schiller GJ, Kletzel M, Ho PJ, Vichinsky E, von Kalle C, Cavazzana M, Leboulch P, Petrusich A, Soni S, Thompson AA. Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305…. 57th American Society of Hematology Annual Meeting and Exposition. Orlando, FL Dec 5-8, 2015. Blood 126(23); 201.
  7. Winstead M, Walters M. Immunologic reconstitution in patient receiving hematopoietic stem cell transplantation for hemoglobin disorders: single-institution experience. ASPHO 28th Annual Meeting. Phoenix, AZ; May 6-9, 2015. Pediatric Blood & Cancer 62(S2); (Poster).
  8. S Soni, F Boulad, MJ Cowan, S Edwards, J Dahake, and MC Walters. Combined Umbilical Cord Blood and Bone Marrow from HLA-identical sibling donors for Hematopoietic Stem Cell Transplantation in children with Non-malignant disorders, Ped Blood and Cancer 2014; 61:1690-4.
  9. John E. Wagner, Jr., M.D., Mary Eapen, M.B.B.S., Yanli Wang, Ph.D., Kirk R. Schultz, M.D., Donna Wall M.D., Nancy Bunin, M.D., Colleen Delaney, M.D., Paul Haut, M.D., David Margolis, M.D., Edward Peres, M.D., Michael R. Verneris, M.D., Mark C. Walters, M.D., Adam Mendizabal, PhD., Mary M. Horowitz, MD and Joanne Kurtzberg, M.D. for the BMT-CTN. Randomized Trial Comparing Single versus Double Umbilical Cord Blood Transplantation for Childhood Leukemia and Myelodysplasia, N Engl J Med 2014, 371:1685-94.
  10. Suh JH, Kanathezhath B, Shenvi S, Guo H, Zhou A, Tiwana A, Kuypers F, Ames BN and Walters, MC. Metabolomic profiling implicates GSH dysregulation in early experimental GVHD, Plos One, 2014 ; 2014 Feb 18;9(2):e88868.
  11. Karafin, MS, S Graminske, Erickson P, Walters MC, Scott E, Carter S, and A Padmanabhan. Evaluation of the Spectra Optia Apheresis System for Mononuclear Cell (MNC) Collection in Mobilized and Non-mobilized Healthy Donors: Results of a Multicenter Study, J Clin Apher 2014, 29:273-80.
  12. S Soni, F Boulad, MJ Cowan, S Edwards, J Dahake, and MC Walters. Combined Umbilical Cord Blood and Bone Marrow from HLA-identical sibling donors for Hematopoietic Stem Cell Transplantation in children with Non-malignant disorders, Ped Blood and Cancer 2014; 61:1690-4.
  13. Z Romero, F Urbinati, S Geiger, AR Cooper, J Wherley, ML Kaufman, RP Hollis, S Senadheera, R Ruiz de Assin, A Sahagian, A Gellis, XWang, D Gjertson, S DeOliveira, P Kempert, S Shupie, H Abdel-Azim, MC Walters, HJ Meiselman, RB Wenby, T Gruber, V Marder, TD Coates, and DB Kohn. Transduction of Human Bone Marrow CD34+ Cells by a Lentiviral Vector Carrying a Modified Human β-Globin Gene for Sickle Cell Disease, J Clin Invest, 2013 Jul 1. pii: 67930. doi: 10.1172/JCI67930. [Epub ahead of print].
  14. F Locatelli, *NN Kabbara, A Ruggeri, A Ghavamzadeh, I Roberts, C-K Li, F Bernaudin, C Vermylen, JH Dalle, J Stein, R Wynn, C Cordonnier, F Pinto, E Angelucci, G Socie, E Gluckman, *MC Walters, and *V Rocha on behalf of Eurocord and European Blood and Marrow Transplantation (EBMT) group. Outcome of patients with haemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling, Blood 122:1072-78, 2013.
  15. NR. Kamani, MC. Walters, S Carter, et al. Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of a phase II STUDY from the Blood and Marrow Transplant clinical trials network (BMT CTN), Biol Blood Marrow Transpl., 18:1265-72, 2012.
  16. NR. Kamani, MC. Walters, S Carter, et al. Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of a phase II STUDY from the Blood and Marrow Transplant clinical trials network (BMT CTN), Biol Blood Marrow Transpl., 18:1265-72, 2012.