Early Career Researchers Offer Glimpses Into Their Research During Data Blitz at The Saban Research Institute Science Day 2024

Explaining a complex research project to a packed auditorium in five minutes is no easy feat, but it’s one that the poster session finalists were more than ready to take on at The Saban Research Institute Science Day 2024 at Children’s Hospital Los Angeles.

CHLA researchers presented 118 research projects on posters displayed in the Anita S. Watson Courtyard of The Saban Research Building. Many of the presenting researchers were postdoctoral fellows who worked on their projects with their CHLA scientific mentors.

While the theme of this year’s Science Day was “Long COVID in Pediatrics: Implications for Individual and Population Health Outcomes,” the poster session research projects explored a wide range of topics spanning five different categories of science: Basic Science, Clinical Research, Medical Education Research, Community Research and Quality Improvement Research.

A total of 118 research projects were included in the poster session, and 3 reviewers scored each project. Projects with the highest scores in each science category were then passed onto the Poster Session Award Committee, made up of Committee Chair Anoopindar Bhalla, MD, and Committee Members Shana Adise, PhD, and Christopher Kuo, MD. Drs. Bhalla, Adise and Kuo confirmed these 11 poster session finalists.

Following the poster session, Science Day attendees returned to the auditorium for the Data Blitz, an afternoon session in which each poster session finalist was given five minutes to deliver an oral presentation on their project.

Data Blitz presenters

Marian A. Castro
Behavioral Research Associate
Training and Research to Empower NeuroDiversity (TREND) Laboratory
Mentor: Jessica M. Schwartzman, PhD

“Investigating Psychiatric Service Utilization and Mental Health Outcomes in Youth with Autism”

Castro’s project aimed to examine psychiatric service utilization among 105 autistic youth and test the differences in anxiety and depressive symptoms between youth who received no services, those who were given medications, those who received therapy, and those who took part in combined services.

Castro and her collaborators concluded that there was a high utilization of psychiatric services in the autistic youth population, with more severe depressive symptoms recorded among those who received no services and those who received combined services. There was no significant difference in anxiety symptom severity across the different groups. Castro ended her presentation by providing a glimpse of her next steps. “Right now, we’re aiming to conduct a longitudinal study here at CHLA to examine youth with autism across time so we can have a better idea of how psychiatric services utilization may impact their mental health outcomes,” she said.

Samantha Hurwitz
PhD Student and USC Research Assistant
Yong-Mi Kim Laboratory
Mentor: Yong-Mi Kim, MD, PhD, MPH

“Inhibition of TBL1 and β-catenin Interaction Sensitizes Acute Lymphoblastic Leukemia to Chemotherapy”

Hurwitz’s project investigated relapse in acute lymphoblastic leukemia (ALL), and the role of Transducin beta like 1 (TBL1), a key component of a pathway that supports the renewal of leukemia-initiating cells when TBL1-binds to β-catenin. Using tegavivint, a novel small molecule inhibitor that prevents β-catenin from binding to TBL1, Hurwitz and her colleagues were able to show that tegavivint in combination with chemotherapy helped to eradicate ALL cells and prolong survival. “In conclusion, ALL is sensitive to TBL1/β-catenin inhibition using tegavivint, and targeting TBL1 could be beneficial for ALL patients,” she explained.

Indulaxmi Seeni, MD
Pediatric Resident and George Donnell Society for Pediatric Scientists Scholar
Department of Anesthesiology Critical Care Medicine
Mentor: Anoopindar Bhalla, MD

“Extra-pulmonary Organ Dysfunction in Pediatric Acute Respiratory Distress Syndrome”

The goals of Dr. Seeni’s project were to determine whether extrapulmonary organ dysfunction phenotypes in children with pediatric acute respiratory distress syndrome (PARDS) can be identified and whether these phenotypes are associated with mortality in the ICU, length of mechanical ventilation, or ventilator-free days. Dr. Seeni and her collaborators concluded that two newly characterized phenotypes are associated with a greater risk of mortality in PARDS than a third phenotype. Also, as Dr. Seeni explained, “the association between extrapulmonary organ dysfunction and mortality appears to be stronger than the association between extrapulmonary organ dysfunction and length of mechanical ventilation.”

Frances Gonzalez, MBS
Clinical Research Coordinator
Center for Endocrinology, Diabetes and Metabolism
Mentor: Jennifer Raymond, MD, MCR

“Group Telehealth Education for Latinx Families and Youth Living with Type 1 Diabetes”

Gonzalez’s work explored providing virtual care as a possible solution to address the obstacles to quality care and worse outcomes experienced disproportionately by Latinx families and youth who are living with Type 1 diabetes.

Through group telehealth sessions, Gonzalez’s project aimed to increase the use of diabetes technology like continuous glucose monitors (CGM) and insulin pumps, decrease patients’ levels of Hemoglobin A1c, expand nutritional knowledge and social support for families, and present diabetes education in a culturally sensitive manner. The telehealth sessions were presented in Spanish, with popular Latinx foods used as examples. Gonzalez and her project collaborators concluded that these virtual groups are a feasible way to boost support for Latinx families, communication is a key tool to maintain family engagement, and additional strategies are needed to address other obstacles to care that these families face. She was optimistic about future research in this area, given these results. “I think of this as an opportunity to learn what else we can do to support these families beyond telehealth,” she said.

Chandra Kaladhar Vemula, PhD
Postdoctoral Research Fellow
Cancer and Blood Disease Institute
Mentor: JinSeok Park, PhD

“Effect of Pax3-Foxo1 Fusion Gene on Cell-Extracellular Matrix Interaction of Rhabdomyosarcoma”

Dr. Vemula’s project investigated the cell-extracellular matrix interaction in rhabdomyosarcoma, which Dr. Vemula describes as “a highly malignant pediatric soft-tissue sarcoma.”

Rhabdomyosarcoma can be split into two subtypes: Fusion protein-positive rhabdomyosarcoma (FPRMS) and fusion protein-negative rhabdomyosarcoma (FNRMS). FNRMS has an increased cell-extracellular matrix interaction compared with FPRMS, and this project sought to explore how different features between the two subtypes could potentially guide the use of distinct approaches to treatment. Ultimately, Dr. Vemula and his fellow researchers concluded that the PAX3-FOXO1 gene was the distinguishing factor among FPRMS and FNRMS, leading to distinct features between these two subtypes. This suggests that different treatments may be effective for the two subtypes.

Hasmik Soloyan, MD
California Institute for Regenerative Medicine (CIRM) Postdoctoral Research Fellow
GOFARR Laboratory for Organ Regenerative Research and Cell Therapeutics in Urology
Mentor: Sargis Sedrakyan, PhD

“Mitochondrial Dysfunction in Glomerular Endothelial Cells and the Role of Extracellular Vesicles in Alport Syndrome”

Dr. Soloyan investigated how fatty acid synthase (FASN) downregulation is involved in glomerular endothelial dysfunction in the kidney disease known as Alport syndrome, as well as how extracellular vesicles that are derived from human amniotic fluid stem cells help to prevent endothelial damage. “While the root cause of Alport syndrome is very well established, mechanisms of disease progression are not fully understood,” she explained.

Dr. Soloyan and her collaborators determined that glomerular endothelial dysfunction is associated with the downregulation of FASN and additional lipid metabolic genes in Alport syndrome. They also gained a deeper understanding of how the function of glomerular endothelial cells and the modulation of the extracellular vesicle-induced metabolic pathway could guide targeted therapies for Alport syndrome and additional chronic kidney diseases.

Meryl Vedrenne-Cloquet, MD, PhD
Postdoctoral Research Fellow
Robinder Khemani, MD, MSCI, Lab
Mentor: Robinder Khemani, MD, MSCI

“Novel Respiratory Phenotypes in Ventilated Children Based on Airway Occlusion Pressure (P0.1) and Esophageal Pressure”

This project aimed to explore new phenotypes depending on airway occlusion pressure (PO.1) and esophageal pressure in ventilated children who have PARDS, and to describe the risk factors for situations in which PO.1 might underestimate high respiratory effort. Dr. Vedrenne-Cloquet and her colleagues determined that in children, PO.1 underestimates high effort quite frequently, and the risk of underestimation appears to increase over time. “Our next step is to now investigate the impact of high effort, drive and phenotypes on the patient’s clinical outcome,” she added.

Madeleine Hare
Clinical Research Coordinator
Kids with Neurogenetic and Developmental Disabilities (KiNDD) Lab
Mentor: Charlotte DiStefano, PhD

“Access to Augmentative and Alternative Communication Among Families of Minimally Verbal Children with Autism Spectrum Disorder”

As 30 to 40% of those with autism spectrum disorder (ASD) have little to no spoken language, this project explored the use of Augmentative and Alternative Communication (AAC) among families with children who have ASD within the CHLA population. The objective of the project was to determine if a family’s home language is related to their access to AAC systems.

Using an anonymous survey, Hare and her fellow researchers gathered information and concluded that families of minimally verbal children with ASD who speak a language other than English in the home could have reduced access to AAC when compared with families who speak only English. “Currently, as part of the second aim of this research study, qualitative interviews are being performed with caregivers, which will give insight and a much richer understanding of parents’ perspectives on accessing AAC and barriers that they face,” Hare explained.

Meaghan E. McCoy
PhD Student and USC Research Assistant
Levitt Lab
Mentor: Anna Kamitakahara, PhD

“Effects of Early-Life High-Fat Diet on Vagal Satiety and Reward Signaling”

McCoy’s project sought to help build an understanding of the mechanisms behind satiety and reward signaling from a developmental perspective, as well as if these mechanisms can be impacted by early dietary experience. “As most of us know, overconsumption of fatty and sugary foods is a major contributor to lifetime disease risk,” McCoy said. The research conducted by McCoy and her colleagues indicated that an early life high-fat diet is associated with increased weight, and enhanced lipid consumption and preference later in life, with more extreme effects observed in females. These results were shown to be the same even if individuals returned to a normal, healthy diet later in life.

Sasha Torres
Research Trainee
Center for Endocrinology, Diabetes and Metabolism
Mentor: Anna Ryabets-Lienhard, DO

“Evaluating the Frequency, Characteristics, and Severity of Non-ossifying Fibromas in Children with Genetic Forms of Rickets at Three Tertiary Care Centers in the U.S. and Canada”

This research looked at non-ossifying fibromas (NOF), which are benign, fibrous lesions of the skeleton that have been observed among children with various forms of the disease known as rickets. Since little is known about how and why NOF occurs in children with rickets, this project aimed to evaluate the frequency and characteristics of NOF in a variety of genetic forms of the condition. Torres and her collaborators determined that NOF occurring in children with genetic forms of rickets could be clinically significant, and that there may be a relationship between NOF and rickets severity. “But more studies are needed to evaluate if severe osteomalacia and/or rickets predispose to NOF development,” Torres said.

Rana Muhammad Shoaib, PhD, PharmD
Postdoctoral Research Fellow
Mahato Laboratory
Mentor: Biraj Mahato, PhD, MS

“Rescuing Vision in a Mouse Glaucoma Model Using Small Molecule Cocktail”

This research focused on developing a potential therapy for optic nerve diseases including glaucoma and optic nerve hyperplasia, conditions that lead to irreversible blindness due to the loss of retinal ganglion cells (RGCs). In vivo RGC reprogramming from resident Müller cells, a type of retinal glial cell, would be a potential method to treat these diseases. Dr. Shoaib’s research demonstrated a small molecule cocktail that can effectively induce endogenous RGC reprogramming from Müller cells, helping to rescue vision in preclinical animal models. Dr. Shoaib highlighted, “This could be a potential treatment for patients with glaucoma and it can overcome challenges associated with pluripotent stem cell therapy.”

And the Winners Are …

Following the Data Blitz presentations, audience members voted for their favorite project, determining the winner of the People’s Choice Award. The rest of the awards were decided by judges Peter Chiarelli, MD, DPhil, Jesse Berry, MD, Michael A. Schumacher, PhD, Mimi Kim, MD, MSc, Nhu Tran, PhD, RN, CCRN, CCRP, Cynthia Cisneros, PhD, MPH, Susan Wu, MD, and Joyce R. Javier, MD, MPH, MS.

Dr. Kuo announced the best poster award winners:

• Basic Laboratory Science Research: A tie between Samantha Hurwitz and Rana Muhammad Shoaib, PhD, PharmD
• Clinical Research: Meryl Vedrenne-Cloquet, MD, PhD
• Community Research: Madeleine Hare
• People’s Choice Award: Meaghan E. McCoy

While closing out the Data Blitz session, Dr. Kuo said, “We hope you all enjoyed this event, learned from your fellow researchers, and developed relationships and greater understandings of what researchers at CHLA are contributing to their field, and most importantly, the patients that this research impacts.”