ResearCHLA 2020 Highlights
Jeffrey Gold, PhD, was interviewed by The New York Times about his research on the use of virtual reality to mitigate pain in children receiving medical care.
Scientific American and various national news outlets reported on the research findings of Michael Goran, PhD, and clinical observations of Rohit Kohli, MBBS, MS, regarding the increase of fatty liver disease in Latino youth.
Research conducted by Laura Perin, PhD, Stefano Da Sacco, PhD, and Roger De Filippo, MD, in the GOFARR Laboratory led to development of a new scientific model that was described in Nature Communications. The model, which filters blood just like human kidneys do, represents a significant step forward in kidney disease research. Their work will support a personalized medicine approach to chronic kidney disease and could aid in testing of new drugs prior to clinical use.
Associated Press reported on research by Arthur Olch, PhD, and Kenneth Wong, MD, about the use of specialized software that could advance treatment accuracy for pediatric cancer patients.
London’s Financial Times quoted Guy Young, MD, on his use of emicizumab to successfully treat children with hemophilia A with factor VIII inhibitors.
The Wall Street Journal carried the story, reported on in the 2018 issue of this magazine, of patient Michelle Lowry and her battle against a life-threatening tumor using a targeted cancer therapy called larotrectinib. The treatment was administered as part of a phase 1 trial by Leo Mascarenhas, MD, MS, and Rachana Shah, MD, of the Cancer and Blood Disease Institute.
Aaron Nagiel, MD, PhD, of The Vision Center, was interviewed on ABC’s Nightline about his treatment of two patients with an inherited form of vision loss. Using this revolutionary FDA-approved gene therapy, Dr. Nagiel was able to successfully deliver the treatment and significantly improve the vision of his patients.
Denise Al Alam, PhD, was recognized by the National Heart, Lung, and Blood Institute—part of the National Institutes of Health (NIH)—and the American Thoracic Society as an investigator who exemplifies the future of the field. Dr. Al Alam was honored for her innovative research of childhood lung diseases such as pulmonary hypoplasia and bronchopulmonary dysplasia.
The NIH’s National Cancer Institute has granted Jesse Berry, MD, $1.14 million to develop a safe method of biopsy for retinoblastoma. Unlike most cancers, traditional tumor biopsy in retinoblastoma is prohibited, as it can spread tumor cells outside the eye. Dr. Berry’s work shows that tumor-specific genetic markers can be found in fluid removed from the front of the eye prior to administering localized chemotherapy. This biofluid can be used as a “surrogate” or liquid biopsy for retinoblastoma. (See page 12 to read more about Dr. Berry’s research.)
The Maurice Marciano Family Foundation Emergency Department and Trauma Center has been named a Pediatric Emergency Care Applied Research Network site, the first federally funded pediatric emergency medicine research network in the United States. Children’s Hospital Los Angeles is one of only 18 hospitals to receive this designation. Todd P. Chang, MD, MAcM, is the site principal investigator on this multiyear infrastructure grant, which provides immediate access to federally funded studies for acutely ill or injured children at CHLA.
The NIH’s National Cancer Institute awarded $1.7 million to David Cobrinik, MD, PhD. Dr. Cobrinik is studying how mutations in the RB1 gene cause cone cells in the retina to proliferate and form retinoblastoma tumors in children.
CHLA’s West Coast Consortium for Technology and Innovation in Pediatrics (CITP), a pediatric medical device accelerator, was awarded $6.6 million from the Food and Drug Administration. One of CTIP’s projects, led by Juan Espinoza, MD, FAAP, is a real-time, prospective data platform that collects clinical data, device data and patient-reported outcomes for pediatric patients with type 1 diabetes. This data platform will then be used for clinical, research and regulatory activities.
Mark Frey, PhD, was awarded $1.7 million from the NIH’s National Institute of Diabetes and Digestive and Kidney Diseases for his study, which will investigate the role that receptor tyrosine kinases ErbB4 and ErbB3 play in the repair and regeneration of the lining of the intestine. The intestinal epithelium is often injured as a side effect of radiation or chemotherapy, or by conditions such as inflammatory bowel disease.
The NIH’s National Institute of Diabetes and Digestive and Kidney Diseases granted $1.7 million to Michael Goran, PhD, Director of CHLA’s Diabetes and Obesity Program, to examine how human milk oligosaccharides affect development of the infant gut microbiome—and how this in turn might predispose children to a wide variety of diseases, such as obesity, and affect cognitive development. He also will explore how extended breastfeeding may protect against obesity in infants. (See page 42 to learn more about Dr. Goran’s research.)
Tracy Grikscheit, MD, was awarded nearly $1.3 million by the California Institute for Regenerative Medicine for her project, which will determine whether using universal pluripotent stem cells derived from adult cells can successfully treat liver failure resulting from metabolic diseases. If successful, this would be a viable alternate option to organ transplant, which is the only currently available treatment for metabolic liver disease.
The NIH’s Eunice Kennedy Shriver National Institute of Child Health and Human Development has awarded Marco A. Hidalgo, PhD, a grant of $3.4 million to longitudinally examine gender identity and mental health among prepubescent children who are transgender or gender nonconforming (TGNC). This is the first federally funded study to establish a large cohort of prepubescent TGNC youth (and their parents). Using surveys, data will be collected at renowned multidisciplinary gender clinics housed within four of the nation’s leading children’s hospitals, including Children’s Hospital Los Angeles.
Mia Humphreys, MSW, was awarded $1.45 million from the Centers for Disease Control and Prevention for her project, which will establish and maintain safe and supportive environments in schools—especially for youth at highest risk of HIV infection and other sexually transmitted diseases (STDs). The project intends to improve the health and well-being of our nation’s youth by reducing HIV, STDs, teen pregnancy and related risk behaviors among middle and high school students.
Yong-Mi Kim, MD, PhD, MPH, received $1.2 million from the NIH’s National Cancer Institute to study drug resistance in leukemia. Dr. Kim was the first to discover that integrin alpha 4 anchors leukemia cells in the bone marrow, allowing them to become resistant to treatment. Her project aims to uncover other integrins that work in concert to shield leukemia cells from chemotherapy, with the eventual goal of devising treatments that can impede this activity and make leukemia cells more sensitive to treatment. (See page 40 to read more about Dr. Kim’s research.)
The NIH’s National Institute of Mental Health awarded $3.7 million to Children’s Hospital Los Angeles Chief Scientific Officer Pat Levitt, PhD, to study the role of the MET receptor in the development of neural circuits. He is studying how the wiring of the brain regions that underlie cognition and social and emotional behavior is built over time.
Irene Lim, LCSW, was granted $2.7 million from the Substance Abuse and Mental Health Services Administration. Her project will expand and enhance comprehensive treatment, early intervention and recovery support services for adolescents and emerging adults with substance abuse who are identified through local high schools and CHLA’s Emergency Department.
Jennifer Raymond, MD, MCR, developed Team Clinic, a shared medical appointment model for adolescents with type 1 diabetes. CHLA received a $1.3 million grant from the Leona M. and Harry B. Helmsley Charitable Trust. In Team Clinic, adolescents meet with their providers one-on-one before attending a patient-driven, shared medical appointment with their peers that allows them to ask questions and receive information in a way they feel is more supportive. The grant will include the development of a virtual toolkit to allow for replication of the model at other institutions.
Wei Shi, MD, PhD, was awarded $2.6 million from the NIH’s National Heart, Lung, and Blood Institute for his study on the disease mechanisms of lymphangioleiomyomatosis (LAM), a rare lung disease. He developed the first genetically engineered mouse with spontaneous LAM-like lung pathology, providing a novel platform for preclinical therapeutic investigation.
The California Community Foundation granted $1.2 million to Arlene Schneir, MPH, Division Administrator for Adolescent and Young Adult Medicine, to promote standardized substance use screening at five Los Angeles Unified School District Wellness Centers located in South Los Angeles. This grant is a partnership between Children’s Hospital Los Angeles, the L.A. Trust for Children’s Health and four federally qualified health centers operating Wellness Centers on these school campuses.
David Warburton, OBE, DSc, MD, MMM, FRCP, FRCS, FRCPCH, Director of the Developmental Biology and Regenerative Medicine Program, has been awarded the rank of Fellow of the American Physiological Society. He is honored for his leadership and pioneering research in the basic and clinical physiology of child health and human developmental biology.
The NIH’s Eunice Kennedy Shriver National Institute of Child Health and Human Development awarded Tishya Wren, PhD, a $2.3 million grant to study bone loss associated with cerebral palsy (CP) in ambulatory children and adults. Study results will help determine who is at highest risk for developing osteoporosis, and will guide the future design of interventions aimed at maximizing and maintaining bone strength in people with CP.
The Alfred E. Mann Family Foundation has dedicated $6 million in support of leading-edge research and clinical care in the fight against childhood cancers and other diseases. Half of this generous gift established the Alfred E. Mann Family Foundation Chair in Cancer Research, and Alan S. Wayne, MD, Director of the Cancer and Blood Disease Institute, is the inaugural chairholder. The additional $3 million supports a new zebrafish research facility led by James Amatruda, MD, PhD, a physician-scientist renowned for developing zebrafish models to discover and test novel therapies for cancer and other diseases.
Oussama Abousamra, MD, joined the Jackie and Gene Autry Orthopedic Center. He earned his doctorate from the University of Damascus in Syria before completing three fellowships in pediatric orthopedic surgery at Nationwide Children’s Hospital in Ohio, Nemours/Alfred I. duPont Hospital for Children in Delaware, and Johns Hopkins University in Maryland. Dr. Abousamra’s clinical and research expertise is in the areas of pediatric neuromuscular and gait disorders, skeletal dysplasia and pediatric limb deformity reconstruction.
James Amatruda, MD, PhD, joined the Cancer and Blood Disease Institute as Endowed Chair of Sarcoma Research and Director of the Alfred E. Mann Family Foundation Zebrafish Facility. He earned his medical degree and doctorate of philosophy in cell biology through the Medical Scientist Training Program at Washington University School of Medicine in St. Louis. Dr. Amatruda’s primary research and clinical focus is on pediatric solid tumors; his lab developed zebrafish models of genetic subtypes of pediatric solid tumors to help identify and validate novel therapies.
Beth A. Carter, MD, joined the Division of Gastroenterology, Hepatology and Nutrition as Medical Director of Liver Transplant and Intestinal Rehabilitation. She provides leadership for the multidisciplinary liver transplant and TPN (total parenteral nutrition) programs. Her research focuses on pediatric liver disease and novel therapeutics for short bowel syndrome patients who are dependent on home TPN. Dr. Carter came to Children’s Hospital Los Angeles from Texas Children’s Hospital in Houston.
Mallory Chavannes, MD, MHSc, joined the Division of Gastroenterology, Hepatology and Nutrition as an attending physician. She earned her doctorate from McGill University in Montreal and completed an advanced fellowship in inflammatory bowel disease (IBD) at the University of British Columbia. Dr. Chavannes’ research focuses on evaluating IBD therapies and finding noninvasive ways of monitoring disease activity in children with IBD.
Christopher Denton, MD, joined the Cancer and Blood Disease Institute as an attending physician specializing in hematological disorders. A recipient of the Hoag Foundation Fellowship Training Grant, his research concentrates on the effect of alpha-globin gene expression on small-vessel blood flow. He earned his doctorate from Oregon Health & Science University before completing his pediatric residency at Seattle Children’s Hospital and his hematology-oncology fellowship at Children’s Hospital Los Angeles.
Michael Goran, PhD, joined the Center for Endocrinology, Diabetes and Metabolism as Director of the Diabetes and Obesity Program. He previously served as Director of the USC Childhood Obesity Research Center. Dr. Goran’s research is focused on dietary treatment of fatty liver disease in the Hispanic pediatric population. He also is investigating how breast milk composition impacts development of the infant gut microbiome as well as weight gain and cognitive outcomes in the first years of life.
Marco A. Hidalgo, PhD, joined the Division of Adolescent and Young Adult Medicine and the Center for Transyouth Health and Development, where he conducts psychotherapy and research. He came from Lurie Children’s Hospital of Chicago, where he co-established the nationally renowned Gender and Sex Development Program for transgender/gender-expansive youth and their families. Dr. Hidalgo’s National Institutes of Health-funded research focuses on identity development and mental health among transgender and gender-nonconforming youth, as well as HIV prevention among sexual and gender minority adolescents.
Kenneth Illingworth, MD, joined the Jackie and Gene Autry Orthopedic Center as a pediatric spine surgeon and Director of the Orthopedic Trauma Program. He earned his doctorate from the University of Tennessee and completed his advanced training in pediatrics and pediatric spinal deformity at Children’s Hospital Los Angeles. Dr. Illingworth specializes in pediatric spinal deformities and has a special interest in pediatric skeletal trauma in patients who have reached or are near skeletal maturity.
Tania Mitsinikos, MD, joined the Division of Gastroenterology, Hepatology and Nutrition. She earned her doctorate at the Keck School of Medicine of USC before completing her internship, residency and fellowship at Children’s Hospital Los Angeles. As Medical Director of the Fatty Liver Clinic, she helps coordinate multidisciplinary research and care for patients with obesity-related nonalcoholic fatty liver disease (NAFLD). Dr. Mitsinikos’ research involves identifying noninvasive biomarkers for children with NAFLD and using nutritional interventions to improve and potentially reverse the disease.
Babak Moghimi, MD, joined the Cancer and Blood Disease Institute after completing his second year of an advanced fellowship in blood and marrow transplantation and immunotherapy at Children’s Hospital Los Angeles. He earned his doctorate at Tehran University of Medical Sciences in Iran, followed by postdoctoral training in cell therapy and a clinical residency in pediatrics at the University of Florida, and a pediatric hematology-oncology fellowship at Children’s Hospital of Philadelphia. Dr. Moghimi conducts leading-edge research in the field of chimeric antigen receptor (CAR) T-cell therapy for neuroblastoma and other childhood cancers.
Diana Moke, MD, MS, joined the Cancer and Blood Disease Institute after conducting a third year of research as a recipient of the National Cancer Institute Ruth L. Kirschstein National Research Service Award Institutional Research Training Grant. She earned her doctorate from the Warren Alpert Medical School at Brown University and conducted her internship and residency training in internal medicine/pediatrics at the University of California, San Diego, before completing her hematology-oncology fellowship training at Children’s Hospital Los Angeles. In her research, Dr. Moke is examining survival trends and related outcomes of adolescents and young adults with cancer.
Bruce R. Pawel, MD, joined the Department of Pathology and Laboratory Medicine as Division Chief of Anatomic Pathology. He has 35 years of clinical, academic and research experience, including the last two decades at Children’s Hospital of Philadelphia, where he most recently served as Pathology Director of the Center for Childhood Cancer Research Biorepository. Dr. Pawel is an internationally renowned expert on the pathology of sarcomas, neuroblastoma and other solid tumors, and his research focuses on identifying potential targets for treating these cancers in children.
Jose Pineda, MD, MSCI, joined Children’s Hospital Los Angeles as Chief of the Division of Critical Care Medicine. He previously served as an Associate Professor of Pediatrics and Neurology at Washington University in St. Louis, Missouri, and as Director of the Neurocritical Care Program at St. Louis Children’s Hospital. Internationally recognized for his research in pediatric neurocritical care, Dr. Pineda is committed to improving treatment and outcomes for children with traumatic brain injuries.
Caitlin Sayegh, PhD, joined the divisions of General Pediatrics and Adolescent and Young Adult Medicine as a psychologist specializing in treating adolescents. She earned her doctorate in clinical psychology at the USC Dana and David Dornsife College of Letters, Arts and Sciences before completing her postdoctoral fellowship at Children’s Hospital Los Angeles. Dr. Sayegh’s research focuses on psychotherapy change processes and mobile health interventions to improve medication adherence among youth with chronic medical conditions.
Saranya Veluswamy, MD, joined the Cancer and Blood Disease Institute as an attending physician after completing her third year of hematology-oncology training as a recipient of the Hoag Foundation Fellowship Training Grant. She earned her doctorate from the The Tamil Nadu Dr.M.G.R. Medical University in India. Her research focuses on the relationship between blood flow, pain and nervous system regulation in children with sickle cell disease.
Alaina Vidmar, MD, joined the Center for Endocrinology, Diabetes and Metabolism after completing her fellowship in pediatric endocrinology at Children’s Hospital Los Angeles. She did her medical training and completed her residency in pediatrics at Children’s Hospital of Wisconsin. Dr. Vidmar is investigating the clinical and cost efficacy of mobile health interventions compared with traditional multidisciplinary in-clinic models for pediatric obesity management.
Rongfu Wang, PhD, joined the Cancer and Blood Disease Institute as Co-Leader of the Tumor Microenvironment Program and Director of the Cell Immunotherapy Program. He holds the Endowed Chair in Cell Therapy Research at CHLA. Dr. Wang earned his doctorate from the University of Georgia and most recently served as Director of the Center for Inflammation and Epigenetics at Houston Methodist Research Institute. His research interests include cancer antigen discovery, cancer immunotherapy, innate immune signaling and epigenetic reprogramming.
