Nephrology Research

Katherine Twombley, M.D.

  • Cure Glomerulonephropathy (CureGN) - Sponsored by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), and NephCure Kidney International for the study of minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), membranous nephropathy (MN), and immunoglobulin A nephropathy (IgAN), which aims to improve the diagnosis, treatment and outcomes of patients affected by these diseases. This study has a large biobank and database of pediatric patients that a fellow can use to develop a research project.
  • Nephrotoxic Injury Negated by Just-In-Time Action (NINJA) - Sponsored by the Agency for Healthcare Research and Quality (AHRQ). QI study to test a new approach to spread a medication safety intervention (‘NINJA’) and reduce more harm by planning for critical contextual and implementation factors during scale-up. Using routine surveillance of medications, lab monitoring, and changes in medication regimens, NINJA reduced nephrotoxic medication related Acute Kidney Injury (NTMx-AKI) by 62% at one hospital. In an AHRQ-funded prospective mixed methods study, four hospitals did not reduce AKI at all while five hospitals reduced AKI by over 50%.
  • Improving Renal Outcomes in Transplantation (IROC) - IROC is a network-based learning health system of patients and families, clinicians, and researchers from the most advanced pediatric institutions in the country. This collaborative works together to solve difficult problems for children with kidney disease by sharing best practices and data regarding care, applying quality improvement methods, and engaging in research to improve health outcomes.

David Selewski, M.D.

  • Prepare-NS - FDA funded grant to develop a patient reported outcomes standard reporting tool for the FDA to use in patients with nephrotic syndrome and fluid overload.
  • Nephrotic Syndrome Study Network (NEPTUNE) study - MUSC is a participating site in NEPTUNE. The NEPTUNE study is collaborative investigational infrastructure of over 25 North American sites for conducting clinical and translational research in Focal Segmental Glomerulosclerosis (FSGS), Membranous Nephropathy (MN), and Minimal Change Disease (MCD). NEPTUNE provides prospective, un-blinded, standardized evaluation of clinical and molecular outcomes in two cohorts: 1) adults and children with incident, biopsy-proven FSGS, MCD, or MN, recruited at the time of first biopsy, and 2) children with incident NS without a diagnostic kidney biopsy (Nephrotic Syndrome Study Network - NEPTUNE (
  • Pediatric Nephrology Research Consortium (PNRC) - Dr. Selewski is the site lead for this multicenter consortium. The PNRC currently includes over 100 participating sites and is dedicated to supporting research in pediatric nephrology. Their primary goals include the development and execution of multi-center retrospective and prospective clinical and translational studies addressing important clinical problems faced by nephrologists. The PNRC also is dedicated to providing opportunities for pediatric nephrology fellows and junior faculty to participate in multi-center clinical and translational studies and publications to enhance their career development (Pediatric Nephrology Research Consortium - Home (
  • Neonatal Kidney Collaborative (NKC)Dr. Selewski is one of the founding members and currently sits on the steering committee for the Neonatal Kidney Collaborative (NKC - Neonatal Kidney Collaborative - Official Site | This multicenter (> 35 cites) multidisciplinary group is dedicated to the multicenter study of neonatal critical care nephrology and neonatal acute kidney injury. This research group has published the seminal work in neonatal AKI, the Assessment of Worldwide Acute Kidney Injury Epidemiology (AWAKEN) study in neonates (Incidence and outcomes of neonatal acute kidney injury (AWAKEN): a multicentre, multinational, observational cohort study - PubMed ( This consortium remains very active in research, mentoring, education, and advocacy.
  • Prospective pediatric acute kidney injury research group (ppAKI NEXUS) - This multicenter consortium is dedicated to the study of critical nephrology in children. This research group is multidisciplinary with expertise in pediatric nephrology, pediatric critical care, and pediatric cardiac critical care. This group published the seminal work evaluating the epidemiology and impact of acute kidney injury in critically ill children, the Assessment of Worldwide Acute Kidney Injury & Renal Angina Epidemiology (AWARE) study (Epidemiology of Acute Kidney Injury in Critically Ill Children and Young Adults - PubMed (
  • Kidney Interventions During Membrane Oxygenation (KIDMO) study group - Dr. Selewski was founding member and former chair of this multicenter research consortium dedicated to studying acute kidney injury, fluid overload, and continuous renal replacement therapy in children on extracorporeal life support. This group remains active and has published over 10 publications of their multicenter work on the topic.
  • Neonatal and Pediatric Heart and Renal Outcomes Network (NEPHRON) study - The Neonatal and Pediatric Heart and Renal Outcomes Network study is a multicenter, retrospective cohort study of consecutive neonates less than 30 days dedicated to understanding the incidence and impact of acute kidney injury and fluid overload in infants undergoing heart surgery for congenital heart disease. This study just published its first two papers from a multicenter cohort of over 2000 infants undergoing congenital heart surgery including the definitive work evaluating the incidence and impact of acute kidney injury in this population (Epidemiology of Acute Kidney Injury After Neonatal Cardiac Surgery: A Report From the Multicenter Neonatal and Pediatric Heart and Renal Outcomes Network - PubMed (

Oana Nicoara, M.D.

  • Standardizing Care to Improve Outcomes in Pediatric Endstage Kidney Disease (SCOPE) collaborative - Collaborative prevents infections in children and adolescents on peritoneal dialysis and hemodialysis using large-scale collaboration to identify and spread effective interventions across pediatric care settings. SCOPE is recognized as evidence-based, data-driven, effective, and methodologically robust by leading organizations and is affiliated with the Children’s Hospital Association. There is also a new partnership with Nationwide Children's to test the role of AMPs and other components of our immune system as biomarkers of peritonitis in patients receiving chronic peritoneal dialysis that fellows can participate.

Omar Moussa, Ph.D. and Katherine Twombley, M.D.

  • Translational Research - Transplant renal allografts do not last forever, with the most common cause of renal allograft loss being antibody mediated rejection (AMR). Currently, the only way to detect transplant renal antibody allograft injury is by measuring serum creatinine, donor specific antibodies (DSA), and doing a renal allograft biopsy, but biopsies are invasive and timely. Urine is a highly desirable median for the study of biomarker analysis because it can be easily collected multiple times by non-invasive techniques in significant volumes in any age patient. Urine contains cellular components, biochemicals, and proteins that reflect the allograft’s metabolic and pathophysiologic state at the time it is collected. Unfortunately, there is a dearth of information about the urine proteome/peptidome. Exosomes offer a potential viable solution. Exosomes are extracellular vesicles that are secreted by cells in times of stress or activation of the cell and can be found in a wide range of biological fluids including urine. Exosomes contain content that represent the state of the secreting parent cell, so examining them can give valuable information about what is going on in tissue and could provide the same information as a biopsy noninvasively.

Denis Guttridge, Ph.D.

  • Basic Science Research - Dr. Guttridge is the director of DCRI and is exploring mechanisms of cachexia in cancer patients and is looking at a cytokine called GDF15. There’s two Ab against this cytokine (one against the ligand, the other against the receptor) that’s in clinical trial for cachexia. We’re testing the Pfizer antibody in our mouse model. He is now interested in exploring whether circulating levels of GDF15 can serve as a biomarker in kidney disease as we think it can in cancer cachexia. This could also be a marker of rejection in kidney transplant patients as there there’s a high level of inflammation, you’ll find elevated levels of GDF15.

Dieter Haemmerich, Ph.D.

  • Basic Science Research - Liposomes have been employed as cancer therapy clinically since the 1990s, with the primary benefit of reduced toxicity but no appreciable efficacy improvement. Thermosensitive liposomes (TSLs) are specifically formulated such that they release the encapsulated drug when exposed to hyperthermic temperatures in the fever range (~40-42°C) and have been investigated as cancer therapy for several decades, with first clinical trials initiated in the last decade. Combined with localized hyperthermia, TSLs allow precise drug delivery to a targeted region. Typically, the targeted tissue is exposed to localized hyperthermia facilitated by an image-guided hyperthermia device. Thus, TSLs enable image-guided drug delivery where drug is delivered to a tissue region identified by medical imaging. This therapy has a lot of potential for rejection in organ transplantation. Currently, when a patient has rejection of the graft, they get systemic anticoagulation which has systemic side effects including infections. This therapy would allow direct delivery of medication to the graft without the dangerous side effects.

Takako Makita, Ph.D

  • Her lab studies genetic and molecular mechanisms that control innervation of kidneys during embryogenesis, which could serve as a potential research project for the Pediatric Nephrology fellows. Her research addresses molecular mechanisms that control establishment of the peripheral nervous system (PNS). A precise PNS circuitry is essential not only for neonatal survival but also for postnatal development and function throughout life. Furthermore, how the nervous system becomes correctly wired remains one of the major unexplained mysteries of biology. My lab uses mouse genetics and physiological assessment to dissect the biological roles of several signaling pathways in peripheral neural development, coupled with molecular and biochemical approaches to understand the underlying mechanisms in each process. All of these have direct application to human disease. Some of the current major projects include:
  1. Sympathetic axon guidance to the heart, which is relevant to arrhythmia
  2. Migration and fate specification of neural progenitors that populate the intestinal enteric nervous system, which is relevant to the congenital neurological disorder Hirschsprung disease
  3. Synapse formation that influences glomerular morphogenesis and function in the kidney, which is relevant to kidney disease and renal transplant
  4. Fate specification of neurons in the inner ear, which is relevant to congenital deafness Waardenberg-Shah syndro