Child Neurology Foundation Shields Research Grant
Supports translational or clinical research by a child neurologist or developmental pediatrician early in his/her academic career. The selected investigator will receive a $100,000 grant of $50,000 per year for two years. The Shields Grant is fully supported by the Winokur Family Foundation and the Pediatric Epilepsy Research Foundation (PERF).
- The applicant must be a junior faculty member who has developed clinical research skills and has a plan for further development of that research or has basic science research skills related to child neurology and who has a plan to translate the new knowledge into clinical care for children with neurologic diseases.
- The Shields Grant must have a clinical research /patient care component.
- The applicant is a legal resident of the United States or Canada
- The applicant is a Junior or Active member of the Child Neurology Society.
- Candidate are NOT disqualified if they have received NIH funding.
- A pre-application is no longer required.
Procedure and Application for Dodge Award and Shields, LGS, and PERF Grants available March 1, 2016 at http://cns.nonprofitcms.org/a/Solicitations/Home/1085.
2015 Shields Research Grant Recipient: Patricia Musolino, M.D.
Massachusetts General Hospital/Harvard Medical School
“Brain Endothelial Dysfunction in Adrenoleukodystrophy”
Cerebral Adrenoleukodystrophy (CALD) is an inherited devastating disease where inflammatory cells infiltrate the brain and cause progressive degeneration that leads to vegetative state or death in months to years. Unfortunately, current therapies either fail to prevent cerebral disease or carry high toxicity and mortality. The support provided by the Child Neurology Foundation Shields Grant will allow me to study how the gene defect changes brain vessel permeability allowing access of inflammatory cells to the brain using imaging in patients and laboratory tools at the bench. More specifically, the study will probe the effect of the gene (ABCD1) deficiency upon blood brain barrier integrity at both the tissue and molecular level by (1) Applying new MRI tequnique to determine if changes in permeability occur prior to lesion progression in patients with CALD (Aim 1) and; (2) Evaluating in-vitro the effects of lack of ABCD1 upon the barrier function of human brain endothelial cells (Aim 2). If validated by this study, this approach sets forth a successful strategy to: (1) identify which patients are at risk of developing cerebral disease; (2) monitor and improve current treatments and; (4) develop an assay to screen for novel therapeutic targets. Results obtained will also help me to prepare future clinical research studies as well as an application to an NINDS Exploratory Trials R01. My passion for patient care and commitment to rigorous science ensures that my research will embrace new ideas and technologies in a highly interdisciplinary environment.
2014 Shields Research Grant Recipient: Joana Osorio, M.D.
University of Rochester
“Cell-based therapy for Pelizaeus-Merzbacher disease”
This research project aims to develop a cell-based treatment strategy for Pelizaeus-Merzbacher disease (PMD), a severe pediatric disorder of myelin caused by mutations in the proteolipid protein gene (PLP1). By transplanting genetically corrected cells from affected patients in a murine model of PMD, I will test their ability to rescue the phenotype and produce normal myelin. I will use induced pluripotent stem cells from patients with duplications and missense mutations in the PLP1 gene, correct the mutations by using gene-editing techniques and subsequently differentiate those to oligodendroglial fate. After intracerebral transplantation in a murine model of PMD, I will evaluate their motor performance and posteriorly the histology of engrafted cells. If this study is successful, it will provide a proof of principle that autologous cell transplantation can be a feasible strategy for treatment of congenital disorders of myelin.
My career goal as a clinician-scientist is to bridge basic research and clinical work in order to establish new treatment strategies for pediatric white matter disorders for which no cure is presently available. The Shields Award represents an important step towards this goal, by supporting my research project that will focus on Pelizaeus-Merzbacher disease (PMD). Success of this pre-clinical study will advance clinical translation of cell-based therapies that can be applied not only to PMD but potentially to other leukodystrophies.
2013 Shields Research Award: Hannah Tully, M.D.
Seattle Children’s Hospital/Seattle Children’s Research Institute
“Pathogenesis and Clinical Implications of Primary Pediatric Hydrocephalus”
One in a thousand newborn infants is diagnosed with hydrocephalus before they even leave the hospital, which can leave their families blindsided. My research proposal seeks to address basic questions about hydrocephalus in children: why it happens, how best to treat it, and what it means for a child’s future. There are three parts to my project: First, I will use MRI-based techniques to explore the relationship between the shape of a child’s brain and the way that cerebrospinal fluid and blood flow within and around it. Second, I will collect detailed clinical information about how children with different types of hydrocephalus develop physically and cognitively, and how they respond to various types of surgery. Finally, I will use these results to guide genomic investigations of the factors that give rise to different types of hydrocephalus, and to differences in clinical outcome. The goal of my work is a deeper understanding of why hydrocephalus develops, a better grasp of its clinical implications, and a new sense of how to tailor treatment to each individual child.