About

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.

Millicent, Mentor and Inspiration

04/17/2019

Photo of Millicent MurrayA stalwart of the CBMFC program, Millicent has been a Program member since 2011 and Fiscal Manager since 2015. We all know she is a hardcore Eagles fan, but not everyone knows about the true breadth of Millicent’s interests beyond her responsibilities to the CBMFC and Peds Hem/Onc Fellowship programs:  CHOP Bioresponse Team member, CHOP Multicultural Professional Network member and Spark Mentor.

About Our Center/History

02/12/2019

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

02/05/2019

The Babushok laboratory seeks an enthusiastic and dedicated Ph.D. or M.D./Ph.D. scientist to join our laboratory as a Post-Doctoral Fellow at the Perelman School of Medicine University of Pennsylvania. The Babushok laboratory performs cutting-edge translational research at the interface of hematology, immunology and molecular genetics to interrogate the mechanisms of acquired and inherited bone marrow failure and the link between bone marrow failure and leukemia. We recently identified a critical connection between the development of clonal hematopoiesis in immune-mediated bone marrow failure called aplastic anemia and somatic loss of specific HLA class I alleles, shedding light on the mechanism of the autoimmune attack in this disease. Research opportunities are available investigating various facets of the immune mechanism of aplastic anemia. The studies will utilize a variety of cutting-edge proteomics and immunogenetic approaches, including mass spectrometry-based proteomic analyses of immune epitopes, next-generation sequencing analyses of HLA and TCR genes, use of genetic engineering technologies to create cell lines bearing designer TCR and HLA alleles, CRISPR-based cell culture models, cytotoxicity assays, RNA interference and pharmacologic inhibition of involved pathways using in vitro and murine models.

Research Topic: murine models of bone marrow failure

Postdoctoral Fellow Opening

02/04/2019

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

02/03/2019

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

Research Topic: Bone Marrow Failure, Clonal Evolution, Hematopoiesis

Olson Clinical Trial #2

02/02/2019

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

Research Topic: Bone Marrow Failure

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

01/30/2019

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.

Research Topic: Bone Marrow Failure

Olson Clinical Trial #6

01/30/2019

Eltrombopag Study (#17-014047, NCT03025698) A Phase II, open-label, non-controlled, intra-patient dose-escalation study to characterize the pharmacokinetics after oral administration of Eltrombopag in pediatric patients with refractory, relapsed or treatment naïve severe aplastic anemia or recurrent aplastic anemia (Novartis)

Research Topic: Bone Marrow Failure

Olson Clinical Trial #5

01/30/2019

TransIT Study (#16-013471, NCT02845596) Unrelated Donor Transplant versus Immune Therapy in Pediatric Severe Aplastic Anemia (Pulsiper/Williams, North American Pediatric Aplastic Anemia Consortium)

Research Topic: Bone Marrow Failure

Olson Clinical Trial #4

01/30/2019

Telomere diseases SCT Trial (IRB 17‐014074, NCT01659606): Radiation‐ and alkylator‐free hematopoietic cell transplantation for bone marrow failure due to dyskeratosis congenita / telomere disease (Agarwal, Boston Children’s Hospital)

Research Topic: Bone Marrow Failure