About Our Center/History
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:
- To provide world-class comprehensive clinical care for patients and families with inherited and acquired forms of bone marrow failure
- To initiate and incite groundbreaking basic, translational, and clinical research to improve diagnosis and treatment for patients with BMF.
At the core of the CBMFC’s activities, Dr. Bessler established the CHOP/PENN Bone Marrow Failure Data Registry and Sample Repository (CHOP IRB# 10-007569), which enables our patients, through consent to use their tissue samples and de-identified clinical data, to participate in research discoveries that enable the next generation of treatments and cures for patients with BMF disorders. Since its inception, this Registry/Repository has grown to include over 650 unique patients/families and over 10,000 samples. This resource has enabled the publication of numerous manuscripts and conference proceedings, as well as the acquisition of federal and foundation funding to conduct cutting edge research dedicated to BMF.
After establishing the CBMFC, Dr. Bessler recruited Dr. Daria Babushok M.D./Ph.D., Dr. Helge Hartung M.D., and Dr. Timothy Olson M.D./Ph.D., along with a number of talented scientists, students, and coordinators, to join her in conducting research and providing clinical care. Following Dr. Bessler’s retirement in 2015, Drs. Babushok, Hartung, and Olson have continued to expand the scope of both the research and clinical activities of the CBMFC. With the 2018 addition of Dr. Peter Kurre, M.D. as Director, the CBMFC continues to thrive, producing innovative basic research, conducting groundbreaking clinical trials, and providing outstanding clinical care as one of the few truly combined Adult/Pediatric centers for the care of patients with BMF conditions in the world.