Research Track 1: Discovery-based Pediatric Research
Andrew Landstrom, MD, PhD, (Track Lead, Duke) leads a research program dedicated to identifying novel genetic and molecular mechanisms of cardiomyopathic and channelopathic disease, uncovering the molecular basis of nodal arrhythmias and junctional tachycardias to inform novel drug design, and clarifying the diagnostic relevance of incidentally identified genetic variants.
Barbara Savoldo, MD, PhD, (Track Lead, UNC) has contributed to the development of new vectors and targets for chimeric tumor-specific T-cell-directed therapeutics (CAR) and the Advanced Cellular Therapies facility that generates modified T-cells for human use.
Gowthami Arepally, MD, (Duke) studies immune thrombocytopenias, thrombotic disorders, and applications of therapeutic apheresis for immunologic disorders, with current projects investigating the role of complement activation in the immune pathogenesis of heparin-induced thrombocytopenia.
Aravind Asokan, PhD, (Duke) uses directed evolution to generate targeted viral vectors for preclinical and clinical gene therapy applied to muscular dystrophy, cardiac failure and neuropathies.
Eric Benner, MD, PhD, (Duke) focuses on improving the survival and quality of life of high-risk neonates in the NICU, through investigating the most common form of perinatal brain injury, white matter injury, and the underlying mechanisms involving neural stem cells and oligodendrogenesis.
Toni Darville, MD, (UNC) studies the relationship between Chlamydial infection and immune responsiveness in adolescent girls and young women, with important implications for vaccine development.
Ian Davis, MD, PhD, (UNC) studies the relationship between chromatin and transcriptional regulation in cancer and development, using chromatin-based strategies, genomic approaches and genome-wide computational analytics for fundamental mechanistic studies, small molecule screening and drug discovery, and for the development of novel diagnostics.
Stephen Frye, PhD, (UNC) conducts collaborative translational research, with a broad background in drug discovery and development, having co-discovered a marketed product (Avodart) and established a GSK department that discovered two FDA-approved kinase inhibitor oncology therapies (Lapatinib and Pazopanib).
Rasheed Gbadegesin, MD, MBBS, (Duke) explores the genetic basis, pathogenesis, and determinants of variable therapy response in nephrotic syndrome (NS), contributing to the identification of novel genetic causes of kidney diseases and establishing a biorepository pediatric NS samples.
Tim Gershon, MD, PhD, (UNC) uses mouse modeling to explore pediatric brain tumors as disorders of developmental brain growth by defining the molecular controls of brain growth that become disrupted and targeting these controls using novel therapeutics.
Joseph Heitman, MD, PhD, (Duke) explores molecular and cellular fungal pathogenesis and mechanisms underlying immunosuppressive and antimicrobial drugs with direct relevance to preclinical pediatric therapeutic pipeline.
Ilona Jaspers, PhD, (UNC) is focused on the effect of tobacco use, environmental pollutant exposure, and e-cigarette use on epithelial cell and immune cell function ex vivo as well as in vivo human response to viral infection, using the live attenuated influenza vaccine as an infection model, with special attention to atopic populations.
Tal Kafri MD, PhD, (UNC) is focused on the development and usage of HIV-1 based vectors for gene therapy applications and as a means to study HIV-1, with emphasis on using mouse genetics to identify host factors affecting the efficacy of gene delivery and HIV-1 pathogenesis.
Michael Kastan, MD, PhD, (Duke) has made significant contributions to the field of cancer biology, including discoveries regarding DNA damage and repair, tumor suppressor genes including p53, genetic and environmental causes of cancer, cell cycle checkpoints, and the DNA Damage Response (DDR) pathway, leading directly to important opportunities for novel cancer therapeutics.
Matthew Kelly, MD, MPH, (Duke) works to develop strategies that use targeted manipulation of the microbiome for the prevention and treatment of infections in high-risk groups of children, particularly elucidating the complex relationships that exist between sociodemographic factors, the upper respiratory microbiome and colonization by potential pneumonia pathogens among infants in Botswana
Allan Kirk, MD, PhD, (Duke) an internationally recognized leader in the field of transplantation, explores transplant biology and preclinical development of novel immunosuppressive agents using animal models.
Michael Knowles, MD, (UNC) studies genotype-phenotype relationships in cystic fibrosis and other diseases of the airways with abnormal mucociliary and cough clearance, including primary ciliary dyskinesia (PCD) and COPD.
Corinne Linardic, MD, PhD (Duke) studies rhabdomyosarcoma (RMS), a cancer related to the skeletal muscle lineage and the most common childhood soft tissue sarcoma, using cell and murine models to probe the molecular contributions of mutations to tumorigenesis, treatment resistance and relapse.
Alexandre Rotta, MD, (Duke) investigates novel mechanisms for mechanical ventilation support for pediatric acute respiratory failure and novel support techniques and lung protective strategies, in particular for critical acute viral bronchiolitis.
Mari Shinohara, PhD, (Duke) uses mouse models for infectious and autoimmune diseases to understand the cellular and molecular mechanisms of pathogen recognition by pattern recognition receptors in macrophages and dendritic cells, initiation of inflammatory responses in the innate immune system, and the impact of innate immune inflammation on the development and regulation of T cell-mediated adaptive immune responses.
Mark Zylka, PhD, (UNC) conducts research in neuroscience focused on nociception, pain modulation, and autism, including circadian “clock” genes and their mechanistic role in mammalian circadian rhythms and G protein-coupled receptors in nociceptive or itch-sensing neurons.
Research Track 2: Pediatric First in Human and Clinical Trials
Dwight Koeberl, MD, PhD, (Track Lead, Duke), develops new therapies for glycogen storage diseases (GSDs) using animal models and translates these discoveries into clinical trials; he has used gene therapy to reveal a novel therapeutic target, leading to completion of a Phase I/II study of a β2-agonist drug, clenbuterol, in late-onset Pompe disease.
Michelle Hernandez, MD, (Track Lead, UNC), as Associate Director of Clinical Research for CEMALB, studies airway inflammation and inhaled pollutants, including strategies to monitor and mitigate these responses.
Wesley Burks, MD, (UNC) leads the UNC Food Allergy Initiative and the UNC Children’s Research Institute and focuses on the mechanistic basis and therapeutic strategies for food allergies, including peanut immunotherapy.
Michael Cohen-Wolkowiez, MD, PhD, (Duke) member of the DCRI, Director of Duke Early Phase Clinical Research, and director of the DCRI Pharmacometrics Center, is well-suited to lead the Clinical Trials Track, with a highly successful career in pediatric clinical pharmacology and drug development.
C. Michael Cotten, MD, MHS, (Duke) studies neuroprotection via autologous cord blood cells for infants with hypoxic ischemic encephalopathy, genomic contributions to morbidities of extremely preterm births, new technologies for newborn screening, and the microbiome in metabolism, immune function, and outcomes of preterm infants.
Matt Laughon, MD, MPH, (UNC) leads research on neonatal epidemiology, pharmaco-epidemiology, and clinical pharmacology and was chosen as UPTiC Mentor for his research experience in clinical trials of therapeutics in infants and successful inter-institutional track record of mentorship.
Joseph Muenzer, MD, PhD (UNC) focuses on congenital metabolic disorders such as Mucopolysaccharidoses (MPS), and his research includes developing new avenues for treatment in children with MPS, including clinical trials to test new forms of intravenous enzyme replacement therapy as well as alternative therapies such as gene therapy and intrathecal drug delivery devices.
Scott Palmer, MD, (Duke) conducts basic, translational and clinical research in transplantation and advanced lung disease, with a background including the first human studies on the importance of innate immunity in transplant rejection and a prospective multicenter study improving CMV prevention post transplantation.
Joseph Piven, MD, (UNC) studies the pathogenesis, molecular genetics and neuropsychological basis of autism related disorders, magnetic resonance imaging of early brain development, and clinical studies of autism.
Svati Shah, MD, MHS, (Duke) investigates molecular epidemiology of cardiovascular disease, utilizing diverse genetic and genomic techniques for identification of risk biomarkers and novel mechanisms of disease.
Cynthia Toth, MD, (Duke) is engaged in multidisciplinary research on the evaluation and treatment of vitreoretinal diseases, significantly contributing to the diagnosis and treatment for retinal diseases/conditions in adults and children, including age-related macular degeneration (AMD).
Research Track 3: Real World Evidence/Outcomes in Pediatrics
Jennifer Li, MD, MHS, (Track Lead, Duke), focuses on pediatric clinical trials and outcomes studies involving the cardiovascular system, leading efforts in drug development to improve the dosing, safety, and efficacy of pediatric therapeutics and to develop study methods for pediatric outcomes with unique regulatory experience of translating research findings into medical practice.
Michael D. Kappelman, MD, MPH, (Track Lead, UNC) is a pediatric gastroenterologist and health services researcher focused on clinical and comparativeness effectiveness research in chronic conditions including Inflammatory Bowel Disease.
Ebony Boulware, MD, MPH, (Duke) explores the influence of medical, social, community, and health system factors on chronic illnesses in adults and children such as kidney disease, hypertension, and cardiovascular disease.
Lesley Curtis, PhD, MS, (Duke) uses observational data to answer questions related to health care delivery, comparative effectiveness, safety, and patient-centered outcomes across a broad array of clinical conditions and clinical care settings.
Scott Kollins, PhD, (Duke) is interested in psychopharmacology and the intersection of ADHD and substance abuse, particularly cigarette smoking.
Susanna Naggie, MD, MHS, (Duke) studies the patient outcomes of accelerated liver fibrogenesis in patients with HIV and viral hepatitis, focusing on the interaction of HIV and HCV on host lipid pathways, uses HIV/HCV co-infection as a model to understand immune dysregulation in HIV viral co-infections and pathways of liver disease pathogenesis in this population, and pharmacogenomics markers in HIV/HCV including IL28B and ITPA.
Sean O'Brien, PhD, (Duke) brings unique experience managing and analyzing large multicenter clinical data sets and developing methodology for health services and outcomes research; his work includes comparative effectiveness trials, evaluation of record linkage methods, observational studies with longitudinal and survival endpoints, and complex data analysis.
T. Michael O’Shea, MD, MPH, (UNC) leads epidemiologic studies of brain damage and neurodevelopmental outcome in low gestational age newborns and developmental follow up of participants in neonatal clinical trials.
Bryce Reeve, PhD, (Duke) focuses on enhancing patient-reported outcomes (PROs) in clinical research and improving the quality of care for pediatric and adult cancer patients, including the development of qualitative and quantitative PRO measures and integration of PRO data in research and healthcare delivery
Betsy Sleath, PhD, (UNC) focuses on provider-patient communication about medications and how this impacts adherence and patient-centered outcomes in childhood and adolescence, recruiting patients from diverse socioeconomic, ethnic and racial backgrounds into clinical research studies.
P. Brian Smith, MD, MPH, MHS, (Duke) is an expert in clinical trial design, clinical research, and biostatistics, focusing his career on pediatric pharmacology, drug safety, and outcomes research. He is currently PI for the Coordinating Center for the Environmental Influences on Child Health Outcomes (ECHO).
Til Stürmer, MD, PhD, (UNC) directs the Center for Pharmacoepidemiology, leads a pharmacoepidemiology training program, is a member of the FDA Drug Safety and Risk Management Advisory Committee, and conducts research on methods of nonexperimental treatment comparisons, including comparative effectiveness.
Kanecia Zimmerman, MD, MPH, (Duke) works to improve the safety and efficacy of drug administration in children by using pharmacoepidemiologic methods and clinical trials to evaluate the effects of sedatives and analgesics on pediatric pain and sedation.