Hematology/Oncology Laboratories

Oncology Laboratories

Bone Tumors

 

Nabil Ahmed, MD, MSc 
Clinical and Translational research
The group develops strategies to treat resistant solid and liquid malignancies using engineered immune cells.


 

Meenakshi G. Hegde, MD
Bone tumors, Brain tumors, Cell and Gene Therapy, Ewing sarcoma, Solid tumors


 

Chris Tsz-Kwong Man, PhD 
Translational research
Dr. Man's laboratory is interested in utilizing multi-omics approaches to identify clinically-applicable biomarkers, therapeutic targets, and pathogenic mechanisms in pediatric bone tumors. 

 

 


 

Lisa Wang, MD 
Bone Tumors, Genetics and Genomics
Translational research

Dr. Wang's laboratory is focused on understanding how defects in a specific gene pathway, RECQL4, which is important for maintaining genomic stability in cells, contribute to the pathogenesis of osteosarcoma. The ultimate goal is to find better therapies for osteosarcoma through better understanding of the molecular pathways driving this particular tumor.

Brain Tumors

 

Nabil Ahmed, MD, MSc 
Clinical and Translational research
The group develops strategies to treat resistant solid and liquid malignancies using engineered immune cells.


 

M. Waleed Gaber, PhD
Translational research
Dr. Gaber's research focuses on building preclinical animal models as well as imaging tools and animal cognitive models that would allow us to study the effect of cranial radiotherapy on brain tumors as well as normal tissue sequalae.


 

Meenakshi G. Hegde, MD
Bone tumors, Brain tumors, Cell and Gene Therapy, Ewing sarcoma, Solid tumors

 


 

Stephen Christopher Mack, PhD 
Translational research
Dr. Mack's laboratory seeks to understand the epigenetic basis of pediatric brain malignancies to develop effective therapies and delineate mechanisms of disease pathogenesis.


 

Jed Nuchtern, MD 
Ewing sarcoma, Bone tumors, Liver tumors, Neuroblastoma, Neuro-oncology, Rare tumors, Surgical oncology


 

Donald “Will” Parsons, MD, PhD 
Genetics and Genomics
Clinical and Translational research
Dr. Parsons' current research primarily focuses on the clinical application of genomic technologies in pediatric cancer care. His team is currently engaged in a number of sequencing studies of pediatric brain tumors and other high-risk and rare pediatric cancers with goals of revealing tumor biology, identifying potential molecular targets for therapy, and facilitating pre-clinical characterization of novel therapeutics.

Cancer Survivorship

Philip Lupo, PhD, MPH 
Clinical and Epidemiological research
Dr. Lupo’s laboratory examines the molecular epidemiology of childhood cancer and developmental disorders. Research interests include: 1) understanding the risk of cancer among children with congenital anomalies; 2) characterizing genetic susceptibility to pediatric rhabdomyosarcoma; 3) phenomic and genomic studies of congenital anomalies; and 4) addressing disparities in acute lymphoblastic leukemia susceptibility and outcomes. The ultimate goal of Dr. Lupo’s research is to discover factors that can be used in new prevention efforts and targeted interventions to limit the adverse consequences of pediatric diseases.

Cell and Gene Therapy

Nabil Ahmed, MD, MSc 
Bone Tumors, Brain Tumors, Ewing Sarcoma, Sarcoma
Clinical and Translational research

The group develops strategies to treat resistant solid and liquid malignancies using engineered immune cells.


Malcolm Brenner, MD, PhD 
One interest of Dr. Malcolm Brenner’s research is in the field of tumor immunology. Tumor cells that have been genetically modified to express immunostimulatory genes will induce effective anti-tumor responses in a range of syngeneic animal models.


Alan Davis, PhD 
Dr. Alan Davis’s research interests are in gene targeting/integration using DNA viruses vectors, particularly adenovirus and adeno-associated virus. The use of adenovirus would be for oncology while adeno-associated virus, for cardiovascular disease


Meenakshi G. Hegde, MD
Bone tumors, Brain tumors, Cell and Gene Therapy, Ewing sarcoma, Solid tumors


Ann Leen, PhD 
Lymphoma
Dr. Ann Leen’s research interests involve using cytotoxic T cells (CTL) for the prophylaxis and treatment of virus-associated malignancies. She is also developing methods to allow the generation of CTL lines directed against tumor antigens expressed in lymphoma. 


Leonid Metelitsa, MD, PhD 
Basic, Clinical and Translational research
Through exploring natural and engineered properties of Natural Killer T (NKT) and other innate immune effector cells, Dr. Metelitsa's laboratory is developing and testing novel immunotherapeutic strategies against multiple types of cancer.


Carlos Ramos, MD 
Clinical and Translational research
Dr. Ramos' research focuses on T-cell therapy for cancer, including phase 1 trials of CAR-T cells targeting CD19, CD30 and kappa light chain as therapy for lymphomas; and HPV-specific T cells as therapy for HPV-associated cancers.


Cliona Rooney, PhD 
Bone Marrow/Stem Cell Transplant, Bone Tumors, Neuroblastoma
Translational research
Dr. Rooney's laboratory is developing and performing immunotherapy trials for patients with a variety of diseases. They are using multivirus-specific T-cells for the treatment of viral infections after hematopoietic stem cell transplantation (HSCT). They are using EBV-specific T-cells for the treatment of EBV-associated malignancies occurring outside the HSCT setting and developing modifications of these T-cells to provide additional signaling to enhance their expansion, persistence and anti-tumor activity. They are targeting GD2-positive malignancies such as osteosarcoma and neuroblastoma, using VZV-specific T-cells modified with a GD2.CAR so that GD2.CAR-VZVST expansion and persistence can be enhanced by stimulation via the TCR using the commercially available VZV vaccine. Finally, they are also enhancing GD2.CAR T-cells by expression of a constitutively active IL7 receptor.


Thomas Westbrook, PhD 

Epidemiology

Philip Lupo, PhD, MPH 
Long-Term Survivors
Clinical and Epidemiological research
Dr. Lupo’s laboratory examines the molecular epidemiology of childhood cancer and developmental disorders. Research interests include: 1) understanding the risk of cancer among children with congenital anomalies; 2) characterizing genetic susceptibility to pediatric rhabdomyosarcoma; 3) phenomic and genomic studies of congenital anomalies; and 4) addressing disparities in acute lymphoblastic leukemia susceptibility and outcomes. The ultimate goal of Dr. Lupo’s research is to discover factors that can be used in new prevention efforts and targeted interventions to limit the adverse consequences of pediatric diseases.


 

Michael Scheurer, PhD 
Epidemiology, Long-Term Survivors
Clinical and Epidemiological research
Dr. Scheurer’s laboratory examines risk factors for the development of childhood cancers, poor treatment outcomes in children being treated for cancer, and the development of late effects in survivors of pediatric cancers . In addition, the laboratory strives to understand the genetic, environmental, and social determinants of  health disparities in these outcomes.

Ewing Sarcoma

Nabil Ahmed, MD, MSc 
Clinical and Translational research
The group develops strategies to treat resistant solid and liquid malignancies using engineered immune cells.


Meenakshi G. Hegde, MD
Bone tumors, Brain tumors, Cell and Gene Therapy, Ewing sarcoma, Solid tumors


Jed Nuchtern, MD 
Ewing sarcoma, Bone tumors, Liver tumors, Neuroblastoma, Neuro-oncology, Rare tumors, Surgical oncology


Jason Yustein, MD, PhD> Basic and Translational research
Dr. Yustein's laboratory focuses on understanding the molecular pathogenesis of pediatric sarcoma metastasis and therapeutic resistance. In addition, his laboratory utilizes our extensive inventory of both human and murine sarcoma models, many developed in the laboratory, to perform pre-clinical studies assessing the efficacy of novel therapeutic approaches targeting these high-risk conditions.

Genetics & Genomics

Lisa Bouchier-Hayes, PhD  Genetics and GenomicsBasic research Her laboratory is focused on two major areas: (1) caspase regulation in cancer; and (2) caspase activation during inflammation. The rationale for these investigations is that caspases protect from cancer and other diseases, by engaging diverse cellular mechanisms in addition to cell death, such as cell division, inflammation, and DNA repair. The ultimate goal is to understand how these caspase pathways can be manipulated for preventative and therapeutic purposes.


Donald “Will” Parsons, MD, PhD 
Neuro-Oncology
Clinical and Translational research
Dr. Parsons' current research primarily focuses on the clinical application of genomic technologies in pediatric cancer care. His team is currently engaged in a number of sequencing studies of pediatric brain tumors and other high-risk and rare pediatric cancers with goals of revealing tumor biology, identifying potential molecular targets for therapy, and facilitating pre-clinical characterization of novel therapeutics.


Sharon Plon, MD, PhD, FACMG
Basic, translational and clinical research


 

Pulivarthi Rao, PhD 
The main goal of Dr. Pulivarthi Rao’s laboratory is to analyze the genetic basis of deregulation of cellular proliferation and differentiation associated with malignant transformation.


Lisa Wang, MD 
Bone Tumors
Translational research

Dr. Wang's laboratory is focused on understanding how defects in a specific gene pathway, RECQL4, which is important for maintaining genomic stability in cells, contribute to the pathogenesis of osteosarcoma. The ultimate goal is to find better therapies for osteosarcoma through better understanding of the molecular pathways driving this particular tumor.

Histiocytosis
Leukemia

 

Terzah Horton, MD, PhD 
Basic and Translational research
Dr. Horton's research laboratory is exploring the relationship between protein degradation and response to chemotherapy in pediatric leukemias such as ALL and AML.


 

M. Monica Gramatges, MD, PhD 
The objectives of the Gramatges laboratory are to uncover genetic risks, such as underlying defects in telomere maintenance, that may predispose children to cancer and cancer therapy-related toxicities as well as late effects.


 

Philip Lupo, PhD, MPH 
Epidemiology, Long-Term Survivors
Clinical and Epidemiological research
Dr. Lupo’s laboratory examines the molecular epidemiology of childhood cancer and developmental disorders. Research interests include: 1) understanding the risk of cancer among children with congenital anomalies; 2) characterizing genetic susceptibility to pediatric rhabdomyosarcoma; 3) phenomic and genomic studies of congenital anomalies; and 4) addressing disparities in acute lymphoblastic leukemia susceptibility and outcomes. The ultimate goal of Dr. Lupo’s research is to discover factors that can be used in new prevention efforts and targeted interventions to limit the adverse consequences of pediatric diseases.


 

Debananda Pati, PhD 


 

Karen R. Rabin, MD, PhD 
Translational research
Dr. Rabin's laboratory research focuses on tumor and host factors affecting outcome and toxicities in acute lymphoblastic leukemia (ALL). A major focus of her laboratory research is on ALL in children with Down syndrome, including somatic and germline genomic profiling to investigate leukemia susceptibility and targeted therapies in this population. Other research interests include molecular determinants of prognosis, identification of novel therapeutic targets, and racial and ethnic outcome disparities in childhood ALL. 


 

Michele Redell, MD, PhD 
Translational research
Research in Dr. Redell’s laboratory is focused on determining mechanisms of chemotherapy resistance in pediatric acute myeloid leukemia (AML). Projects include investigations of signaling and gene expression differences between chemosensitive and chemoresistant AML cells, and preclinical investigations of novel therapies to overcome chemoresistance mechanisms.

Liver Tumors

Andras Heczey, MD 
Basic, clinical and translational research
Active projects in the laboratory focus on examining the solid tumor microenvironment components' gene expression and cellular neighborhood characteristics to understand the immuneescape mechanisms and design effective countermeasures. To overcome the metabolic competition between the tumor cells and effector lymphocytes, the lab is examining ways to engineer T cells. The Heczey Lab's ultimate goal is to develop new genetic engineering strategies to reprogram cells inside the human body, in situ.


Sanjeev A. Vasudevan, MD 
Solid Tumors, Liver Tumors
Translational research
Dr. Vasudevan's laboratory focuses on basic and translational research related to pediatric liver cancer. His lab creates unique patient-derived xenografts for small molecule, targeted drug testing and strive to characterize high-risk characteristics such as vascular invasion, chemoresistance, and metastatic spread.

Long Term Survivors

 

M. Monica Gramatges, MD, PhD 
Leukemia
The objectives of the Gramatges laboratory are to uncover genetic risks, such as underlying defects in telomere maintenance, that may predispose children to cancer and cancer therapy-related toxicities as well as late effects.


Philip Lupo, PhD, MPH 
Epidemiology, Solid Tumors, Leukemia
Clinical and Epidemiological research
Dr. Lupo’s laboratory examines the molecular epidemiology of childhood cancer and developmental disorders. His research interests include: 1) understanding the risk of cancer among children with congenital anomalies; 2) characterizing genetic susceptibility to pediatric rhabdomyosarcoma; 3) phenomic and genomic studies of congenital anomalies; and 4) addressing disparities in acute lymphoblastic leukemia susceptibility and outcomes. The ultimate goal of Dr. Lupo’s research is to discover factors that can be used in new prevention efforts and targeted interventions to limit the adverse consequences of pediatric diseases.

Lymphoma

 

Ann Leen, PhD 
Cell and Gene Therapy (CAGT)
Dr. Ann Leen’s research interests involve using cytotoxic T cells (CTL) for the prophylaxis and treatment of virus-associated malignancies. She is also developing methods to allow the generation of CTL lines directed against tumor antigens expressed in lymphoma.

Neuroblastoma

 

Andras Heczey, MD 
Basic, clinical and translational research
Active projects in the laboratory focus on examining the solid tumor microenvironment components' gene expression and cellular neighborhood characteristics to understand the immuneescape mechanisms and design effective countermeasures. To overcome the metabolic competition between the tumor cells and effector lymphocytes, the lab is examining ways to engineer T cells. The Heczey Lab's ultimate goal is to develop new genetic engineering strategies to reprogram cells inside the human body, in situ.


 

Leonid Metelitsa, MD, PhD 
Cell and Gene Therapy
Basic, clinical and translational research
Through exploring natural and engineered properties of Natural Killer T (NKT) and other innate immune effector cells, Dr. Metelitsa's laboratory is developing and testing novel immunotherapeutic strategies against multiple types of cancer.


 

Jed Nuchtern, MD 
Ewing sarcoma, Bone tumors, Liver tumors, Neuroblastoma, Neuro-oncology, Rare tumors, Surgical oncology


 

Cliona Rooney, PhD 
Bone Marrow/Stem Cell Transplant, Bone Tumors, Cell and Gene Therapy, Neuroblastoma
Translational research
Dr. Rooney's laboratory is developing and performing immunotherapy trials for patients with a variety of diseases. They are using multivirus-specific T-cells for the treatment of viral infections after hematopoietic stem cell transplantation (HSCT). They are using EBV-specific T-cells for the treatment of EBV-associated malignancies occurring outside the HSCT setting and developing modifications of these T-cells to provide additional signaling to enhance their expansion, persistence and anti-tumor activity. They are targeting GD2-positive malignancies such as osteosarcoma and neuroblastoma, using VZV-specific T-cells modified with a GD2.CAR so that GD2.CAR-VZVST expansion and persistence can be enhanced by stimulation via the TCR using the commercially available VZV vaccine. Finally, they are also enhancing GD2.CAR T-cells by expression of a constitutively active IL7 receptor.

Rare Tumors

 

Jed Nuchtern, MD 
Ewing sarcoma, Bone tumors, Liver tumors, Neuroblastoma, Neuro-oncology, Rare tumors, Surgical oncology

Retinoblastoma

 

Mary Hurwitz, PhD 
The Hurwitz laboratory studies the use of gene therapy in the treatment of ocular disease. The Hurwitz's are also exploring the use of gene therapy as a treatment for retinal degenerative diseases such as retinitis pigmentosa and macular degeneration.


 

Richard Hurwitz, MD 
The Hurwitz laboratory studies the use of gene therapy in the treatment of ocular disease. The Hurwitz's are also exploring the use of gene therapy as a treatment for retinal degenerative diseases such as retinitis pigmentosa and macular degeneration.

Solid Tumors

Nabil Ahmed, MD, MSc 
CAGT, Brain Tumors, Sarcoma
Clinical and Translational research
The group develops strategies to treat resistant solid and liquid malignancies using engineered immune cells.


Andras Heczey, MD 
Liver tumors, Neuroblastoma
Basic, clinical and translational research
Active projects in the laboratory focus on examining the solid tumor microenvironment components' gene expression and cellular neighborhood characteristics to understand the immuneescape mechanisms and design effective countermeasures. To overcome the metabolic competition between the tumor cells and effector lymphocytes, the lab is examining ways to engineer T cells. The Heczey Lab's ultimate goal is to develop new genetic engineering strategies to reprogram cells inside the human body, in situ.


Philip Lupo, PhD, MPH 
Epidemiology, Long-Term Survivors
Clinical and Epidemiological research
Dr. Lupo’s laboratory examines the molecular epidemiology of childhood cancer and developmental disorders. Research interests include: 1) understanding the risk of cancer among children with congenital anomalies; 2) characterizing genetic susceptibility to pediatric rhabdomyosarcoma; 3) phenomic and genomic studies of congenital anomalies; and 4) addressing disparities in acute lymphoblastic leukemia susceptibility and outcomes. The ultimate goal of Dr. Lupo’s research is to discover factors that can be used in new prevention efforts and targeted interventions to limit the adverse consequences of pediatric diseases.


Meenakshi G. Hegde, MD
Bone tumors, Brain tumors, Cell and Gene Therapy, Ewing sarcoma, Solid tumors


Sanjeev A. Vasudevan, MD 
Solid Tumor Programs, Liver Tumor Program
Translational research
Dr. Vasudevan's laboratory focuses on basic and translational research related to pediatric liver cancer. His lab creates unique patient-derived xenografts for small molecule, targeted drug testing and strive to characterize high-risk characteristics such as vascular invasion, chemoresistance, and metastatic spread.

Surgical Oncology

Jed Nuchtern, MD 
Ewing sarcoma, Bone tumors, Liver tumors, Neuroblastoma, Neuro-oncology, Rare tumors, Surgical oncology


Sanjeev A. Vasudevan, MD 
Solid Tumors, Liver Tumors
Translational research
Dr. Vasudevan's laboratory focuses on basic and translational research related to pediatric liver cancer. His lab creates unique patient-derived xenografts for small molecule, targeted drug testing and strive to characterize high-risk characteristics such as vascular invasion, chemoresistance, and metastatic spread.

Hematology Laboratories

Bone Marrow Failure

 

Alison Bertuch, MD, PhD 
Bone Marrow Failure, Genetics and Genomics
Basic science, translational, clinical
The Bertuch laboratory studies basic mechanisms of telomere maintenance and DNA repair and the genetic and molecular defects in these essential processes that underlie inherited bone marrow failure and cancer predisposition syndromes.

Genetics & Genomics

 

Alison Bertuch, MD, PhD 
Bone Marrow Failure, Genetics and Genomics
Basic science, translational, clinical
The Bertuch laboratory studies basic mechanisms of telomere maintenance and DNA repair and the genetic and molecular defects in these essential processes that underlie inherited bone marrow failure and cancer predisposition syndromes.

Sickle Cell Disease

 

Jonathan Flanagan, PhD 
Hematology, Sickle Cell Disease
Basic and translational research
The Flanagan laboratory research mission is to utilize high-quality genomic studies to better understand the determinants of severe clinical outcomes in patients with sickle cell disease. In particular, they are using next-generation sequencing to identify genes and polymorphisms that impact the risk of stroke in young patients. His laboratory performs sophisticated functional studies to interpret the functional consequences of these natural differences.