Bone Marrow Failure

People

Timothy Olson, MD, PhD

Photo of Timothy Olson, MD, PhD
Principal Investigator

Dr. Olson's clinical efforts focus on pediatric hematopoietic stem cell transplantation (HSCT), with a specific emphasis on HSCT for patients with inherited and acquired bone marrow failure.

Daria Babushok, MD, PhD

Photo of Daria Babushok, MD, PhD
Principal Investigator

Dr Daria Babushok is a physician-scientist and an Assistant Professor of Medicine in the Division of Hematology-Oncology at the University of Pennsylvania. Dr Babushok laboratory conducts basic and translational research focusing on the immune pathogenesis of acquired aplastic anemia and the development of clonal hematopoiesis in acquired aplastic anemia and other bone marrow failure disorders.

Ding-Wen Chen, PhD

Ding-Wen Chen, PhD
Postdoctoral Fellow

Dr. Chen completed his PhD at the University of Waterloo in 2018 with research training at the interface of drug delivery, gene therapy, nanomedicine and regenerative medicine. As a postdoctoral fellow at Kurre’s laboratory, Dr.

Ji Zha, PhD

Ji Zha, PhD
Postdoctoral Fellow

Dr. Ji Zha is a postdoctoral fellow in Dr. Timothy Olson’s laboratory. She is currently studying on the cellular and molecular roles of hematopoietic stem cell (HSC) niche in donor HSC engraftment following hematopoietic stem cell transplantation (SCT) in mouse models of inherited bone marrow failure, including Fanconi Anemia, Shwachman Diamond Syndrome, and Dyskeratosis Congenita.

An Steensels

Photo of An Steensels
Research Scientist

An has a Master of Science in Biology from the University of Leuven in Belgium, and did a master thesis project on the contributions of ARF6 and Presenillins in endosomal trafficking in Alzheimer’s Disease. After graduating in 2015, she lived in Tanzania, Spain, and Belgium, where she worked on several science and animal rescue projects. In addition, An went back to school to get a degree in Veterinary Technology.

Seul Jung

Photo of Seul Jung
Research Technician

Seul Jung received her Bachelors of Science degree in Biology at Temple University in 2019. Her previous research involved sequencing zebrafish to study the effects of mutations in the Pygopus2 gene. She was also involved in research focused on the relationship between invadopodia and cell cycle progression in breast cancer cells. Seul became a Research Technician for Dr.

Melissa Colden

Photo of Melissa Colden
Research Scientist

Melissa focused on public health and nutrition throughout undergrad and spent free time in the chemistry lab. After graduating in 2013, she did volunteer research at the San Francisco VA Medical Center studying the roles of miRNAs in prostate cancer and developed a skillset in molecular biology.

Research

  • 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.

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.

Articles

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

Wednesday, January 30, 2019 Full Article: view

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.

Olson Clinical Trial #5

Wednesday, January 30, 2019 Full Article: view

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

Olson Clinical Trial #3

Wednesday, January 30, 2019 Full Article: view

CHP 894 (IRB 08‐005659, NCT01050855) Reduced Intensity Conditioning (RIC) Regimen for Patients with Non‐Malignant Disorders

Olson Clinical Trial #6

Wednesday, January 30, 2019 Full Article: view

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)

Olson Clinical Trial #4

Wednesday, January 30, 2019 Full Article: view

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)

Olson Clinical Trial #1

Sunday, February 3, 2019 Full Article: view

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 Full Article: view

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