Research

The Comprehensive Bone Marrow Failure (BMF) Center at The Children’s Hospital of Philadelphia brings together physicians and researchers to provide state of the art diagnostics, clinical care and investigational studies as well as basic research focused on acquired and inherited BMF. The center is closely affiliated colleagues caring for adult BMF patients at the Hospital of the University of Pennsylvania.

  • Understand cause of stem cell losses in Fanconi Anemia.
    • Failure of the hematopoietic system with stem cell losses is a near uniform occurrence in FA. The etiology remains unknown and we are using murine models to understand the underlying defect
  • The role of FA proteins during hematopoietic development Hematopoietic development in bone marrow failure diseases is poorly understood and fetal deficits remain to be elucidated.
    • As hematopoietic stem cells undergo maturation to an adult phenotype well after birth, therapeutic opportunities that focus on fetal characteristics may reveal additional therapeutic opportunities.
  • The role of cell-derived extracellular vesicles in stem cell regulation.
    • Cell derived vesicles are constitutively released by cells. The transmission of protein an RNA cargo to other cells in both paracrine and endocrine manner has revealed potent regulatory capacity. We are beginning to study their role in HSC regulation in several loss and gain of function models.

Research Topic: acute myeloid leukemia, Hematopoietic Stem Cells, Fanconi Anemia, Gene Therapy, Extracellular Vesticles

  • The role of germline genetic predisposition syndromes in the development of pediatric myelodysplastic syndrome and acute myeloid leukemia
  • Patterns of HLA allele expression and clonal evolution in acquired aplastic anemia, and how these findings may serve as biomarkers for disease response as well as clues to the underlying immune basis of this disease
  • Investigation of how intrinsic pathways governing hematopoietic stem cell niche function can be harnessed to improve the efficiency and durability of donor engraftment after hematopoietic stem cell transplantation, particularly in the context of bone marrow failure syndromes.
  • Clinical trials of novel conditioning and T cell depletion strategies to improve survival and decrease allo-immune complications for patients with non-malignant hematologic disorders undergoing hematopoietic stem cell transplantation.

Research Topic: Bone Marrow Failure, Hematopoiesis, Aplastic Anemia, clonal hematopoiesis, pediatric myelodysplastic syndrome, acute myeloid leukemia, AML/MDS predisposition, paroxysmal nocturnal hemoglobinuria, Immune evasion, autoimmunity

The long-term goal of the Babushok Laboratory to improve the outcomes of patients with bone marrow failure disorders through bench-to-bedside research aimed at elucidating mechanisms of hematopoietic failure and transforming this knowledge into rational therapies.

Research Topic: Bone Marrow Failure, Hematopoiesis, Aplastic Anemia, clonal hematopoiesis, pediatric myelodysplastic syndrome, acute myeloid leukemia, AML/MDS predisposition, paroxysmal nocturnal hemoglobinuria, Immune evasion, autoimmunity

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About Us

Comprehensive Bone Marrow Failure Center

Graphic of the front of Children's Hospital of PhiladelphiaThe Comprehensive Bone Marrow Failure (BMF) Center at The Children’s Hospital of Philadelphia brings together physicians and researchers to provide state of the art diagnostics, clinical care and investigational studies as well as basic research focused on acquired and inherited BMF. The center is closely affiliated colleagues caring for adult BMF patients at the Hospital of the University of Pennsylvania.

About Our Center/History

Tuesday, February 12, 2019

The Children’s Hospital of Philadelphia/University of Pennsylvania Comprehensive Bone Marrow Failure Center (CBMFC) was established in 2010 by Dr. Monica Bessler, M.D. Ph.D., an internationally recognized Physician-Scientist with expertise in numerous bone marrow failure (BMF) syndromes including dyskeratosis congenita, aplastic anemia, and paroxysmal nocturnal hemoglobinuria. Dr. Bessler defined the mission of the CBMFC as having two goals:

Postdoctoral Fellow Opening

Monday, February 4, 2019

A Postdoctoral position in Hematopoietic Stem Cell Biology is available in the laboratory of Peter Kurre, M.D., at the Comprehensive Center for Bone Marrow Failure. The Laboratory has longstanding expertise in hematopoietic stem cell biology and has actively investigated stem cell losses in Fanconi Anemia (FA) for over a decade. Ongoing work is focused on fetal stem cell losses in murine models of FA and their contribution to the stem cell exhaustion and leukemogenesis that are a hallmark of FA. Investigations will involve a wide range of methods in stem cell and molecular biology, imaging and bioinformatics. The postdoctoral fellow will, under the direction/guidance of a mentor, Peter Kurre, MD, assume responsibility for a specific, on-going research project.

Olson Clinical Trial #1

Sunday, February 3, 2019

CHP 14BT057 (IRB 14‐011465, NCT02928991) Pilot Study of Fludarabine‐based Conditioning for Matched Related Donor Bone Marrow Transplantation in Patients with Acquired and Inherited Bone Marrow Failure Syndromes

Olson Clinical Trial #2

Saturday, February 2, 2019

CHP 16BT052 (IRB 16‐012881, NCT03047746) Unrelated and Partially Matched Related Donor Peripheral Stem Cell Transplant w/ TCRαβ depletion for patients with Bone Marrow Failure Syndromes

Peter Kurre, MD, Appointed Director of the Comprehensive Bone Marrow Failure Center

Wednesday, January 30, 2019

Photo of Peter Kurre, MDPeter Kurre, MD, joined Children’s Hospital of Philadelphia (CHOP) as Director of the Comprehensive Bone Marrow Failure Center on August 31, 2018. In collaboration with bone marrow failure (BMF) experts at the Perelman School of Medicine at the University of Pennsylvania, the center has built an international reputation of excellence in consultation and treatment of patients with BMF disorders, currently following well over 200 patients. Dr. Kurre will lead a world-renowned, multidisciplinary team in developing new therapies for BMF syndromes and in basic research to understand the causes of these syndromes.