Division of Hematology Research
The Division of Hematology at CHOP is committed to several research initiatives, each with a singular goal: Advance the treatment of childhood blood diseases.
We have researchers covering the full spectrum of investigation, from basic research to translational and clinical studies that are advancing patient therapies. Here are some examples of CHOP's latest hematology research:
Sickle Cell Center
The Colket research building, home to the Center for Cellular and Molecular Therapeutics.
Recent and ongoing studies in the Sickle Cell Center are funded by the National Institutes of Health (NIH), the NIH-funded Comprehensive Sickle Cell Center Program, non-governmental funding agencies, corporations, and community organizations and individuals. We are one of 10 Comprehensive Sickle Cell centers in the nation funded on a competitive basis by the NIH's National Heart, Lung and Blood Institute. Our studies include:
- Fetal tolerance, chimerism and sickle cell disease. Hematopoietic stem cell transplantation in utero (IUHSCTx) is a promising approach for the treatment of a variety of hematologic disorders. The goal for in utero stem cell therapy is to treat sickle cell disease before any clinical manifestations of the disease occur. Dr. Alan Flake, a pediatric hematologist and investigator in CHOP's Center for Fetal Research is conducting research that aims to allow for replacement of abnormal blood forming stem cells with normal stem cells, with the hope that eventually, unborn babies treated with this preventative therapy will be born without any syptoms of sickle cell disease.
Center for Cellular and Molecular Therapeutics
The Center for Cellular and Molecular Therapeutics facilitates rapid translation of pre-clinical discoveries into clinical application. One of few such programs based at a pediatric institution, the center collaborates with other major programs to pursue new therapies for inherited and acquired disorders. Katherine A. High, MD, is the director of the Center for Cellular and Molecular Therapeutics at CHOP. Current research includes:
- Gene therapy for hemophilia. Dr. High has used adeno-associated virus (AAV) vectors in clinical trials of gene therapy for hemophilia B. Dr. High also published a study in Nature of genome editing technology in 2011 that was the first successful demonstration of genome editing to correct a genetic defect in vivo.
Pediatric & Adult Comprehensive Bone Marrow Failure Center
CHOP's Pediatric & Adult Comprehensive Bone Marrow Failure Center launched in 2010, representing a major advance in bone marrow diseases. Bone marrow failure, which can be inherited or acquired, is the inability of the bone marrow to produce sufficient circulating blood cells. Monica Bessler, MD, PhD, is founder and director of the Center.
- Molecular and genetic events that lead to bone marrow failure diseases. The goals of Dr. Bessler's research are to refine diagnostic tools and develop more effective and comprehensive treatments. Because blood cells play key roles in fighting infection, carrying oxygen, blood clotting and other important biological processes, better understanding of their production and functions may have medical implications beyond blood disorders.
The Normal and Malignant Hematopoiesis Research Affinity Group
The Normal and Malignant Hematopoeisis Research Affinity Group aims to address problems in gene and protein networks that signal blood cell maturation from primitive bone marrow progenitor cells, as well as networks that regulate the balance of cell growth and cell death. These networks are relevant to hematopoiesis, and understanding more about how they regulate the development of specific blood cell elements in normal and diseased states will allow for development of targeted prevention and more effective treatments for disordered hematopoiesis, leukemia and lymphoproliferative diseases in children.
- Prevention and treatment for various forms of pediatric AML. One form of acute myeloid leukemia (AML), which occurs as a complication of anticancer chemotherapy, has an especially poor prognosis. One project within the Affinity Group is examining how disruption of the cellular protein called topoisomerase II causes chromosomal breakage that results in AML. The goal is to develop safer chemotherapy regimens and protective compounds.
- Reduce complications and mortality from infections associated with intensive AML treatment. GATA-1 is a critical transcription factor protein that programs the expression of genes in blood cell development. The GATA-1 transcription factors are abnormal in the form of AML associated with Down syndrome. Investigators are determining individual genetic variations that underlie infection risk to improve treatment and reduce risk of infection in these patients.