UW to lead national research on pediatric asthma in low-income urban areas
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Above, a child using an asthma inhaler. (Photo: Shutterstock)
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The University of Wisconsin School of Medicine and Public Health has been selected as the national leadership center for a new clinical research network that will conduct pediatric asthma research in low-income urban settings around the country.
The National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, recently announced they have awarded $10 million in first-year funding to establish the clinical research network, called Childhood Asthma in Urban Settings (CAUSE). The NIAID intends to provide approximately $70 million over seven years to support the new network.
The CAUSE Network will be led by principal investigators Daniel Jackson, MD, associate professor, and James Gern, MD, professor, both in the Division of Allergy, Immunology, and Rheumatology. Co-investigators include William Busse, MD, Department of Medicine, and Christine Sorkness, RPh, PharmD, School of Pharmacy, as well as mechanistic collaborators at University of California San Francisco, University of Chicago, University of Washington/Benaroya Research Institute at Virginia Mason, La Jolla Institute, and other clinical research center sites around the country.
“Children who grow up in urban neighborhoods not only have higher rates of asthma but also experience more negative outcomes due to the disease,” said Jackson. “Our previous research efforts have identified some of the variables by which disadvantaged urban environments can increase the risk for allergic diseases and asthma, and CAUSE provides an incredible opportunity to build on these successes and take the next steps toward refining our understanding of asthma and developing therapies that will lessen disease burden and hopefully lead to disease prevention.” Full story.
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Pelin Cengiz, MD, receives Chancellor’s Award for 'Excellence in Research'
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Above, Pelin Cengiz, MD (center), associate professor, Critical Care, talks with researchers in a lab. (Photo: UW Health)
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There are more than 10,000 academic staff on the UW–Madison campus. Each year, the university honors a select group of them for their achievements and dedication to excellence. Pelin Cengiz, MD, associate professor, Critical Care, is one of this year’s recipients and was honored with the prestigious Chancellor’s Award for Excellence in Research (Independent Investigator) during an invitation-only virtual ceremony earlier this month.
In her groundbreaking research, Cengiz studies hypoxic-ischemic brain injury, one of the leading causes of illness and death in children and an area with a paucity of effective treatments. Specifically, she looks at the sex differences in developmental brain injuries of the very young.
Cengiz’s scientific inquiries are informed and motivated by her experiences caring for critically ill children, while her innovative research program addresses one of the most important and understudied areas of children’s health. She is, her colleagues say, the model for what a clinician-scientist should be. Full story.
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A stimulating environment: Waisman Center acquires neuromodulation lab for infants and children
The adaptability of children with disabilities has inspired Bernadette Gillick, PhD, MSPT, PT, associate professor, Developmental Pediatrics and Rehabilitation Medicine, and Waisman Center investigator, for the majority of her career. A physical therapist and neuroscientist, her research focuses on recovery and development in infants who have suffered a stroke between the third trimester in utero and the first month of life.
“Children are so incredibly resilient,” she says. “Coupled with the heightened neuroplasticity of the brain’s ability to rewire itself and to use different areas for functions that we don’t typically think happen in those regions, they’re remarkable.” Full story.
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Pediatric physicians tap biomedical engineering students to design lumbar puncture model
Performing a spinal tap requires precision, particularly when the patient is a newborn. During their residencies, physicians training in pediatrics practice on infant-sized models and observe a live procedure up close. Then it’s their turn—and when that time comes, they’ll feel for the relevant anatomical landmarks, insert a needle and guide it into the spine’s epidural space.
“There is a very big difference between palpating landmarks on a rubber model with plastic bones and actually completing it on a live neonate,” says Dr. Brady Hauser, a resident in the Department of Pediatrics at the University of Wisconsin-Madison.
In an effort to create another training tool for lumbar punctures on infants, Hauser turned to the Department of Biomedical Engineering’s undergraduate design program, which pairs student teams with clients from industry and academia and challenges them to create prototypes of medical devices and technology that address practical, unmet needs. Hauser’s task for the students: create a newborn-sized training simulator that would allow aspiring doctors to practice lumbar punctures under the guidance of ultrasound imaging. Full story.
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Waisman CP Clinic strikes a balance
When twins Sebastian and Charlotte Sundly were born at just 24 weeks, doctors did brain scans on the micro-preemies and noted some bleeds in both infants. That’s when their parents Kendra and Kevin were told their children had cerebral palsy (CP), a group of disorders that affect a person’s ability to move and maintain balance and posture. Because of their diagnoses, the twins have received services at the Waisman Center Cerebral Palsy Clinic since they were less than a year old. The CP Clinic is one of several specialty clinics at the Waisman Center with an interdisciplinary team of clinicians.
“I think it’s really beneficial to have all of the different disciplines available to families for evaluation,” says Melissa Villegas, MD, CP clinical director and assistant professor of pediatrics. “Motor diagnoses can affect children in multiple different ways. Being able to have speech and language pathology, the communication aids, and occupational and physical therapy all together provides a more comprehensive evaluation as well as a cohesive management plan going forward.” Full story.
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‘Dream Team’ recognized with AACR Team Science Award
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Above, pediatric "Dream Team" members (left to right) Drs. Ken DeSantes, Christian Capitini, Paul Sondel, and Mario Otto. (Photo: UW Health)
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The St. Baldrick’s Foundation-Stand Up 2 Cancer Pediatric Cancer Dream Team, which includes pediatric faculty members Christian Capitini, MD, associate professor, Ken DeSantes, MD, professor, Mario Otto, MD, PhD, associate professor, and Paul Sondel, MD, PhD, professor, was selected for the American Association for Cancer Research (AACR)’s Team Science Award. This prestigious award was established by the AACR and Eli Lilly and Company to acknowledge and catalyze the growing importance of interdisciplinary teams to the understanding of cancer. The award serves to recognize an outstanding interdisciplinary research team for their innovative and meritorious science that has advanced our fundamental knowledge of cancer. The Dream Team was chosen in recognition of its collective and significant contributions to the pediatric cancer research community, including the genesis of 183 published manuscripts, submission of 21 patent applications, creation of a new clinical trials network, generation of over $90 million to support innovative research, and for contributing to the treatment of over 950 children on early phase clinical trials.
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James Conway, MD, selected for Dean's Award for excellence in mentorship
 James Conway, MD, professor, Global Health, Infectious Diseases, has been selected for the 2021 Dean’s Award for Excellence in Medical Student Research Mentorship. Conway exemplifies excellence through his leadership in the Global Health program, commitment to mentoring, and his longstanding involvement in student research programs, such as the Shapiro Summer Research Program, the Research Honors Program, and the Path of Distinction in Research. Conway will be presented with the award at Medical Education Day on May 27, 2021. He will also give the Shapiro Guest Lecture at the Medical Student Research Forum on November 22, 2021.
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Paul Sondel, MD, PhD, honored with national clinical and translational science award
 Four University of Wisconsin School of Medicine and Public Health researchers have been honored with prestigious awards from the Association for Clinical and Translational Science (ACTS), in recognition of their excellence and outstanding performance in support of clinical and translational research. Paul Sondel, MD, PhD, professor of human oncology and pediatrics, was given the Edward H. Ahrens, Jr. Award for Outstanding Achievement in Patient-Oriented Research. Sondel is nationally and internationally known for his innovative patient-centered research leading to new standards of care for lethal pediatric cancers. Full story.
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Amy Peterson, MD, and team awarded R21 grant from NIH/NICHD
 Principal Investigator Amy Peterson, MD, associate professor, Cardiology, along with Co-Investigators, Patrice Held, PhD, FACMG, associate professor, Genetics and Metabolism, Vanessa Horner, PhD, Department of Pathology and Laboratory Medicine, and Michael Lasarev, MS, Department of Biostatistics and Medical Informatics, were awarded $427,542 from the National Institutes of Health/Eunice Kennedy Shriver National Institute of Child Health & Human Development (NIH/NICHD) for their project, “Biochemical and molecular newborn screening for familial hypercholesterolemia.” In this study, biochemical and molecular genetic tests will be performed on dried blood spots from Wisconsin newborn screening specimens to design a testing algorithm that can be used to detect familial hypercholesterolemia in the newborn population. With current estimated detection rates at less than 10%, diagnosing individuals in the newborn period has the potential to significantly decrease the burden of heart disease not only to the children identified, but also to their relatives.
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Bikash Pattnaik, PhD, receives grant from Hubble Therapeutics
 Bikash Pattnaik, PhD, assistant professor, Neonatology and Newborn Nursery, received a $106,823 award from Hubble Therapeutics for his project, “Assessment of ocular function in KCNJ13 conditional knockout mice administered subretinal HUB-101 and assessment of expression of HUB-101 in hfRPE cells in monolayer culture.” The project will test HUB-101 expression in cultured human fetal retinal pigment epithelium (hfRPE) cells in vitro. It will also measure the functional recovery of electroretinography (ERG) c-wave, retina structure, and expression of immune markers after subretinal delivery at specified time points in vivo. The goal of HUB-101, a new gene therapy, is to treat the inherited retinal disorder Leber congenital amaurosis, which is caused by mutations in the KCNJ13 gene. Hubble Therapeutics is a biotechnology company which aims to develop a platform of ion channel-targeted therapeutics targeting rare diseases. The data generated from this study will be used to support a clinical trial to evaluate HUB-101 gene therapy.
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Christian Capitini, MD, awarded two UWCCC pilot grants
 Christian Capitini, MD, associate professor, Hematology, Oncology, and Bone Marrow Transplant, was recently awarded two separate grants through the 2020-2021 UW Carbone Cancer Center (UWCCC) Support Grant Pilot Program competition. In the first, Capitini will serve as Co-PI with Lixin Rui, PhD, Department of Medicine, on the $25,000 pilot project, “EGR1 is a new biomarker in aggressive lymphoma.” Capitini will also serve as Co-PI on a second project with Reinier Hernandez, PhD, Department of Medical Physics. They were awarded $50,000 for the project, “Expressing a bispecific Chimeric Antigen Receptor to enable imaging of CAR-T cell trafficking in vivo.”
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Christian Capitini, MD, will collaborate with Saha Lab on new award
Congratulations to Krishanu Saha, PhD, Department of Biomedical Engineering, and Christian Capitini, MD, associate professor, Hematology, Oncology, and Bone Marrow Transplant, who will serve as Co-PIs on a new project funded by the UW Grainger Institute for Engineering. In this one-year, $50,000 seed project, titled “Virus-free biomanufacturing of cell therapies,” the team will develop novel, safe, scalable, virus-free technologies for genome editing within human cells, and ultimately position UW-Madison to be a center for the biomanufacturing of next generation cell therapies.
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David Evans, PhD, awarded NIH grant with Christian Capitini, MD
David Evans, PhD, Department of Pathology and Laboratory Medicine, was recently awarded a new R01 grant from NIH along with Co-Investigator Christian Capitini, MD, associate professor, Hematology, Oncology, and Bone Marrow Transplant. This five-year award (with Year 1 funding in the amount of $764,536), is titled “Harnessing adaptive NK cell transfer to deplete viral reservoirs.” The proposed study, funded by the National Institutes of Health/National Institute of Allergy and Infectious Diseases, will reveal the extent to which viral peptides presented on the surface of HIV-infected cells stimulate natural killer (NK) cell responses through activating receptors. It will provide an important proof-of-concept for the development of NK cell-based therapies to eradicate HIV-1 reservoirs in chronically infected individuals.
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James Gern, MD, awarded U19 opportunity funds from NIAID
 James Gern, MD, professor, Allergy, Immunology, and Rheumatology, was recently awarded U19 Opportunity Funds from the National Institutes of Health/National Institute of Allergy and Infectious Diseases (NIAID) via the University of California-San Diego. His project, titled “AADCRC RNA sequencing core for airway epithelial cells,” is a collaboration with program members Nora Barrett, MD, of Brigham and Women’s Hospital and Steven Ziegler, PhD, of Benaroya Research Institute. The team hypothesizes that combining and analyzing bulk and single cell RNA sequencing data sets from multiple centers will identify a comprehensive epithelial cell transcriptome with multiple uses for AADCRC investigators. The one-year project was funded in the amount of $46,539 total project costs.
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Janet Legare, MD, awarded funding from Johns Hopkins University for achondroplasia study
 Janet Legare, MD, clinical professor, Genetics and Metabolism, was named site Principal Investigator for the study, “A multi-center cohort study of achondroplasia.” The study, part of the Achondroplasia Natural History Study, was awarded $232,951 over three years from Johns Hopkins University and BioMarin Pharmaceutical, Inc. The first phase of this project was a retrospective natural history study involving patients with achondroplasia evaluated at the four academic institutions. The study had four study domains: anthropometry, surgical burden, sleep disordered breathing, and establishment of a radiographic catalogue. In this Phase 2 of the study, efforts to understand the natural history of achondroplasia will continue. The specific aims pertain to craniofacial structure; metabolism, growth and body composition; and pain/physical function related to lower extremity osteotomies and spinal surgery for subjects with achondroplasia.
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Sushant Srinivasan, MD, receives subaward funding from NHLBI
 Sushant Srinivasan, MD, associate professor, Critical Care, was awarded a $7,200 subaward through collaborators from the University of Michigan, and supported by the National Institutes of Health/National Heart, Lung, and Blood Institute (NHLBI). The five-year R01 project, titled “Acute respiratory distress syndrome (ARDS) in children and extracorporeal membrane oxygenation (ECMO) initiation strategies impact on Neuro-Development (ASCEND),” will characterize the one-year outcomes of children supported by EMCO to determine if and when EMCO should be initiated in children with severe lung injury.
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“Dr. Marsh is truly an outstanding role model, provider and team member to all of her learners, patients and colleagues. Her demeanor with families and team members is truly inspiring. She is the type of person with whom you automatically feel safe and secure, and it was amazing to be able to observe her interactions with patients and the strength of their relationships. She is clearly passionate about palliative care and serves an ideal advocate for her patients.”