Clinical Research Activities

Members of the Division of Pediatric Hematology Oncology engage in numerous basic, translational, clinical, medical education, and health disparities research initiatives. Clinical trials include investigator-initiated, consortium, and industry-sponsored initiatives.

Clinical Research Consortia

Beat Childhood Cancer Research Consortium

Beat Childhood Cancer Research Consortium, formerly known as Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC), is a group of 40+ universities and children's hospitals that offer a worldwide network of childhood cancer clinical. These trials are based on the research from a group of closely collaborating investigators who are linked with laboratory programs developing novel therapies for high-risk pediatric cancers. Our mission is to continue to use precision medicine to bring forward new therapies for children with cancer with the goal of finding a cure for these patients.

UCSD/RCHSD PI: William Roberts, MD

Children's Oncology Group (COG)

Children's Oncology Group (COG) is a National Cancer Institute (NCI) supported clinical trials group, is the world's largest organization devoted exclusively to childhood and adolescent cancer research. The COG unites more than 9,000 experts in childhood cancer at more than 200 leading children's hospitals, universities, and cancer centers across North America, Australia, New Zealand, and Europe in the fight against childhood cancer.

Today, more than 90% of 14,000 children and adolescents diagnosed with cancer each year in the United States are cared for at Children's Oncology Group member institutions. COG's unparalleled collaborative efforts provide the information and support needed to answer important clinical questions in the fight against cancer.

The Children's Oncology Group has nearly 100 active clinical trials open at any given time. These trials include front-line treatment for many types of childhood cancers, studies aimed at determining the underlying biology of these diseases, and trials involving new and emerging treatments, supportive care, and survivorship.

The Children's Oncology Group research has turned children's cancer from a virtually incurable disease 50 years ago to one with a combined 5-year survival rate of 80% today. Our goal is to cure all children and adolescents with cancer, reduce the short and long-term complications of cancer treatments, and determine the causes and find ways to prevent childhood cancer.

UCSD/RCHSD PI: William Roberts, MD

Children's Healthcare Advancements in Thrombosis (CHAT)

The Children’s Healthcare Advancements in Thrombosis (CHAT) Consortium is an international collaborative network of more than 69 pediatric institutions across the United States and Australia dedicated to improving the prevention, diagnosis, and management of venous thrombosis in children and young adults. CHAT has led multiple practice-changing studies, including the identification of key risk factors and the development of validated, clinically implemented risk assessment models for thrombosis in children with cardiac disease, critical illness, and central venous catheters. Our overarching mission is to reduce the burden of thrombosis in pediatric patients and optimize long-term outcomes through high-quality research, innovation, and multicenter collaboration.

UCSD/RCHSD PI: Julie Jaffray, MD

Clinical Care Consortium for Telomere Associated Ailments (CCCTAA)

The Clinical Care Consortium for Telomere Associated Ailments (CCCTAA) is an international group of researchers and clinicians dedicated to studying and treating Telomere Biology Disorders (TBDs). Its key functions include fostering collaboration to improve the diagnosis and management of telomere biology disorders, such as dyskeratosis congenita.

UCSD/RCHSD PI: Nicholas Gloude, MD

North American Pediatric Aplastic Anemia Consortium (NAPAAC)

North American Pediatric Aplastic Anemia Consortium (NAPAAC) is a collaborative research effort that seeks to develop better therapies for children with aplastic anemia by combining the expertise and resources of the leading pediatric hematologists in North America.

UCSD/RCHSD PI: Nicholas Gloude, MD

Pacific Pediatric Neuro-Oncology Consortium (PNOC)

Pacific Pediatric Neuro-Oncology Consortium (PNOC) is a network of eight children's hospitals that conduct clinical trials of new therapies for children with brain tumors. Our goal is to improve outcomes by translating the latest findings in cancer biology into better treatments for these children. At PNOC, our focus is personalized medicine – testing new therapies that are specific to the biology of each patient's tumor to maximize their effectiveness. Our goal is to improve overall outcome for children with brain tumors.

UCSD/RCHSD PI: John Crawford, MD, MS

Pediatric Transplantation and Cellular Therapy Consortium (PTCTC)

Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) is the largest clinical trials group focused exclusively on blood and marrow transplants for children and adolescents. Our consortium includes more than 100 pediatric BMT centers in the United States, Canada, New Zealand, and Australia. PBMTC is a core member of the NIH-funded Blood and Marrow Transplant (BMT) Clinical Trials Network (CTN) and has a close collaborative relationship with the Children's Oncology Group (COG). The PBMTC has clinical trials activities focused on the application of BMT for younger patients and works in concert with many of our industrial partners. As the largest forum focused on pediatric BMT, the PBMTC is committed to research that improves clinical outcomes of BMT in children and adolescents. By vigorously advocating for our patients and their families, we strive to increase awareness and resources for all of those who must undergo this life saving treatment.

UCSD/RCHSD PI: Eric Anderson, MD

Pediatric Immune Deficiency Treatment Consortium (PIDTC)

Pediatric Immune Deficiency Treatment Consortium (PIDTC) consists of 42 centers in North America whose shared goal is to improve the outcome of patients with rare, life threatening, inherited disorders of the immune system. Basic scientists, immunologists, and transplant physicians from the participating centers have contributed much of the current knowledge of the cause and treatments of PID. The immediate focus of the consortium is to concentrate on three severe immune disorders which can be cured by hematopoietic stem cell transplantation, enzyme replacement, and/or or gene therapy by bringing together physician/scientists who evaluate and care for the majority of children with PID in North America.

UCSD/RCHSD PI: Eric Anderson, MD

St. Jude Children's Research Hospital/Rady Children's Hospital/UCSD Alliance

This unique alliance is designed to benefit both patient care and research efforts. It enables St. Jude and Rady Children's to combine their resources and expertise to pursue clinical trials and basic and translational research.

UCSD/RCHSD PIs: Deborah Schiff, MD

Basic Science, Translational & Population Health Research

M. Paula Aristizabal, MD, MS

Our clinical research program focuses on understanding pediatric cancer disparities locally and globally and the development of interventions to improve outcomes in racial/ethnic minorities and underserved populations. We study how social determinants of health, such as race/ethnicity, socio-economic status, health literacy, language, and acculturation impact clinical outcomes in children with cancer and blood disorders. Our research methodology centers on implementation science, qualitative research, mixed methods, bioethics and quality improvement.

A. Preethi Ganesan, MBBS, PhD

Our research is focused on understanding the mechanistic basis of robust anti-tumor immune responses and the impact of host genetics and tumor genomics on such responses. Our overall goal is to translate these findings into novel personalized immunotherapy for the treatment of cancer. We utilize a combination of patient-derived tumor samples, mouse tumor models, single-cell sequencing and functional genomics to understand immune events at the level of human tumors and validate their in vivo functional significance.

Shweta Joshi, PhD

The primary goal of our research is to understand molecular mechanisms regulating tumor growth, inflammation and metastasis. Our laboratory is focused on the study of signaling cascades that regulate recruitment and activation of immunosuppressive macrophages in solid tumors with special focus on neuroblastoma and pancreatic adenocarcinoma.

Hojun Li, MD, PhD

The Li lab studies both healthy and abnormal blood cell production. We use these principles as the key foundation for our efforts to solve diseases of child health such as blood disorders and cancer, and to improve the safety and effectiveness of blood and bone marrow transplantation. To achieve these goals, we combine numerous cutting-edge techniques, including single cell multi-omic profiling, machine learning, spectral flow cytometry, CRISPR gene editing, and novel drug target identification for drug repurposing.

Kincheon Bryan Li, MD, PhD

I am a junior faculty member in Raju Lab studying how to circumvent the blood-brain barrier to improve therapies for highly aggressive childhood brain tumors like diffuse intrinsic pontine glioma (DIPG)/diffuse midline glioma (DMG). Specifically, I have been developing novel P-selectin targeting nanomedicines to package anti-cancer therapeutics, enhancing their delivery into the tumor space while sparing normal brain tissue to reduce side-effects. I completed my PhD under Dr. Annie Huang at the University of Toronto and the Hospital for Sick Children, defining the now World Health Organization-recognized molecular subgroups of pineoblastoma, a rare but highly lethal brain tumor arising in young children.

Angela Liou, MD

I am a physician–scientist with a joint appointment at UCSD/Rady Children’s Hospital and Sanford Burnham Prebys Medical Discovery Institute (SBP). My research examines how epigenetic alterations are functionally responsible for driving the development of childhood brain tumors, with a particular focus on pediatric diffuse midline gliomas. In these tumors, expression of the H3K27M oncohistone reshapes the epigenetic landscape in ways that expose actionable dependencies. My overarching goal is to define the mechanisms underlying epigenetic dysregulation and identifying therapeutic vulnerabilities that can be leveraged to inform future preclinical studies and clinical trials for children with CNS tumors.

Praveen Raju, MD, PhD

We are a translational research lab that uses sophisticated mouse genetics approaches to develop improved preclinical models of various pediatric brain cancers to better understand tumor biology as well as for use in therapeutic drug studies. In addition, we use 'state-of-the-art' nanotechnologies to improve drug delivery past the blood-brain barrier with the goal to improve therapeutic efficacy of respective drug payloads and minimize treatment-related toxicities.

Jing Yang, PhD

Our general research interest is to understand the molecular basis of tumor metastasis. We utilize cell and molecular biology tools, mouse tumor models, functional genomics, and 2D/3D imaging techniques to uncover the genes and the signaling pathways responsible for tumor metastasis. Specifically, we have identified two cellular programs, Epithelial to Mesenchymal Transition (EMT) and invadopodia-mediated extracellular matrix degradation as being critical for tumor invasion and metastasis. We continue to dissect the signaling pathways regulating these programs and to test the importance of these signaling pathways in tumor metastasis in both mouse tumor models and human tumor samples.

Helena Yu, MD

I am a junior faculty member conducting research in the Signer Laboratory at the Sanford Consortium for Regenerative Medicine. I investigate the regulation of hematopoietic stem cell (HSC) protein homeostasis (proteostasis) throughout hematopoietic development and in bone marrow failure syndromes. In addition, my research focuses on developing new biomarkers of HSC fitness with the goal of improving HSC transplant outcomes for pediatric cancer patients.

Peter E. Zage, MD, PhD

The primary laboratory projects in the Zage lab include studies to better understand the pathways involved in the regulation of growth factor receptor trafficking and degradation in tumor cells and their role in tumor growth and treatment response in addition to studies to identify novel therapeutic targets and novel agents to use in combination with established therapies.