BRAIN Research Awards 2023

Blood biomarker for ASD: Validation

PROJECT PI: Dwight German, PhD, University of Texas Southwestern Medical Center
PROJECT AIMS: Validate a panel of blood biomarker panel for ASD in a new group of ASD and Typically developing (TD) boys using a comprehensive proteomic and machine learning study.
STUDY DESIGN: Multicenter collaboration to collect serum samples from 100 ASD boys and 100 TD boys (18months-8years) along with demographic, clinical, behavioral, cognitive and genetic data assessments; Proteomic analysis and advanced non-linear Machine learning analysis to identify 8 already studied blood proteins and possible additional proteins as biomarkers.
INNOVATION: Largest proteomic study to validate ASD blood biomarkers, analyzing over 7000 proteins; Multidisciplinary collaboration involving ASD diagnosis, biomarker development and machine learning expertise; Potential blood test for ASD to enable early detection and subtype specific targeted interventions.

Epigenetic mechanisms of innate immune activation in ASD

PROJECT PI: Paul Ashwood, PhD, University of California, Davis
PROJECT AIMS: Understand mechanisms behind increased innate immune activation, through epigenetic mechanisms, in children with ASD and GI comorbidity; Identify potential biomarkers and targets for therapeutic interventions.
STUDY DESIGN: Investigate myeloid cell function in 12 children with ASD and 12 typically developing controls; Investigate epigenetic and immune biological signatures and endotoxin tolerance.
INNOVATION: Possible identification of 8-10 epigenetic biomarkers or markers for macrophage activation in children with ASD comorbid with GI symptoms; Identification of targets for pharmacological interventions; May lead to development of simple, diagnostic tests to identify patient-specific factors to control immune responses specific to ASD associated with inflammatory bowel conditions, that will positively affect behavioral symptoms and quality of life.

Autoantibody and cytokine biomarker discovery in autism

PROJECT PI:  Shannon Rose, PhD. Arkansas Children’s Research Institute
PROJECT AIMS: Explore autoantibodies as potential inflammation markers; Measure levels of inflammatory markers in plasma in patients with Neurodevelopmental Disorders (NDD); Identify potential treatments including anti-inflammatory and immune-modulating therapeutics.
STUDY DESIGN: Analyze samples from Autism Spectrum Disorder (ASD)/NDD patients and controls for 44 different immune factors in plasma; Identify patterns associated with NDD subtypes using statistical and cluster analysis.
INNOVATION: Attempts to address biomarker deficiency in ASD/NDD diagnosis; Novel Integration of CNS autoantibodies and immune factors with detailed patient histories; Potential improvement of diagnostic accuracy and treatments.

Safety and efficacy of tPBM for individuals with autism and epileptiform activity

PROJECT PI: Richard Frye, PhD, Autism Discovery & Treatment Foundation
PROJECT AIMS: To determine the safety of the tPBM with particular focus on its effect on clinical seizures; To determine the efficacy of tPBM for reducing core symptoms of ASD; To determine the efficacy of tPBM for reducing the frequency of the epileptiform activity during wakefulness and sleep as well as the change in delta power (a biomarker for seizure vulnerability) on 24hr overnight video EEG.
STUDY DESIGN: Open label baseline controlled design study in patients with abnormal EEG, with and without epilepsy. Both groups will receive tPBM treatment.
INNOVATION: This study is the first study to specifically investigate the safety and efficacy of tPBM for individuals with ASD and comorbid epilepsy. It will examine both reduction of abnormal epileptiform activity and reduction of active seizures. In addition, it will contribute to the literature on the connection of brainwaves distribution (e.g., power of wakeful Delta waves) and neurological disorders (e.g., ASD) as well as the connection between the distribution of brainwaves (e.g., power of Delta waves) and epileptiform activity as well as active seizures.

Assessing the efficacy of spelled communication

PROJECT PI:  John Gaitanis, MD, Brown Medical School/Hasbro Children’s Hospital Children’s Neurodevelopment Center
PROJECT AIMS: Determine the existence of facilitator bias and dependence in spelled communication; Investigate efficacy of spelled communication in non-verbal patients with ASD; Determine if spelled communication improves behavioral regulation and improves communication and development, thereby the quality of life.
STUDY DESIGN: Follow 25 patients over 1 year through data from questionnaires, interviews and video-recorded responses- Blinded observers will review verbal and spelled responses; Enroll 20 patients 13 years of age and over who have never used spelled communication and randomly assign to spelled or standard therapy; Assess communication ability, changes in behavior and quality of life at 6 and 12 months.
INNOVATION: First ever clinical study to determine if extensive use of spelled communication(rapid prompting, spelling to communicate and S2C) is warranted; First ever retrospective and prospective trials of spelled communication; Assessment of facilitator bias and dependence.

Improving emotion perception in individuals with autism

PROJECT PI: David Whitney, PhD, University of California, Berkeley
PROJECT AIMS: Improve the emotion recognition of individuals with autism using context-based emotion-processing training through visually guided attention; Increase the ability to focus on important social cues during social interactions
STUDY DESIGN: Inferential Emotion Tracking (IET) group training will involve 10 sessions, 5 videos per session, 2-3 sessions; The control group will be given a similar task with gaze patterns from random videos; Gaze-contingent eye tracking will be used to control peripheral vision; Post-training assessments will be performed using IET< facial expression recognition, emotional intelligence and IQ tests.
INNOVATION: Novel paradigm for emotion perception training in autism using context cues in making inferences about others’ emotions; Takes into consideration the advances in eye-tracking research to control peripheral vision; Validity mirroring real-world perception; Potential for improved social functioning and accessible home-based technology interventions.

Mind Over Chatter: Neural decoding of language comprehension in nonspeaking individuals with autism

PROJECT PI:  Laura Gwilliams PhD, Stanford University
PROJECT AIMS: Develop an empirical, passive, neural assessment of language comprehension in non-speaking individuals; Characterize individual “receptive language fingerprints” to measure processing of sounds, words, sentences and meanings.
STUDY DESIGN: Utilize portable EEG system (Emotiv Flex) to record neural responses associated with neural processing as non-speaking autistic individuals listen to an audiobook
Use machine-learning to analyze neural data and quantify 50 aspects of spoken language into 5 feature families; Determine individual “receptive language fingerprints” based on relative strength of feature families; Recruit 30 non-speaking using letterboards and 30 age-matched neurotypical participants.
INNOVATION: First test to understand how non-speaking people process language; Use of benchmark portable-EEG against the gold standard MEG for signal quality; Transformation of research methodologies with a naturalistic task like listening to an audiobook; Advance scientific understanding of receptive language ability in non-speaking autism that can inform development of tailored communication approaches and therapies.

Targeting redox signaling to develop novel interventions for autism

PROJECT PI:  Adrien Eshraghi PhD, University of Miami, Florida
PROJECT AIMS: Determine the contribution of redox signaling in predisposition to ASD following gene-environment (G x E) interaction using a rat model; To develop new preventive therapeutics using repurposed drugs such as FDA approved Edavarone.
STUDY DESIGN: Build on proof-of-concept studies already performed showing rescue of ASD like phenotype in a preclinical rat modelsv; Investigate effects of environmental exposure to phthalates by brain region analysis, histopathologic, transcriptome and pathway analysis; Evaluate redox signaling scavenger effects of Edavarone in ASD-like phenotype in brain tissues; using behavior andsociability assessements, histopathology and fMRI imaging.
INNOVATION: Development of an innovative gene-environment interaction (GxE) pre-clinical rat model to explore influence of genetic factors and environmental exposures like phthalates on development of ASD; Focus on specific molecular pathways involving nitrated proteins and redox signaling ; Testing the use of Peroxynitrite scavenger Edavarone, a readily available FDA approved drug, repurposed as a potential new treatment modality for treating core ASD symptoms

NLRP3 inflammasome activity in ASD: Relationship to oxidative stress status and inhibition by cobinamide

PROJECT PI:  Richard Deth, PhD, Nova Southeastern University
PROJECT AIMS: Understand the connection between inflammation and antioxidant deficits in autism, namely oxidative stress and impaired methylation.; Identify a potential new target for developing a new treatment; Understand the increase in autism numbers and provide new perspectives on autism risk factors and prevalence.
STUDY DESIGN: Investigate the activity of a key inflammation pathway, NLRP3 inflammasome, in the brains of individuals with autism using post-mortem brain samples; Examine the relationship between NLRP3 activity and the body’s antioxidant and methylation status; Evaluate potential of vitamin B12 derivatives – cobinamides, to reduce NLRP3 activity.
INNOVATION: First study to look at the inflammasome activity in the brains of individuals with autism; Use of Cobinamides as a novel therapeutic target for autism.

Studying the role of oxytocin in social attachment with near-infrared oxytocin nanosensors

PROJECT PI: Markita Landry PhD, University of California, Berkeley
PROJECT AIMS: Develop a tool for real-time imaging of oxytocin to study its role in social behaviors; Address the lack of spatial and temporal resolution in existing oxytocin imaging tools
STUDY DESIGN: Generate Nanosensors for oxytocin detection with selectivity over vasopressin, dopamine, GABA and glutamate; Validate nanosensor performance in acute mouse brain slices based on dynamic range, sensitivity and selectivity; Image and measure oxytocin signaling differences in live animals- social and solitary voles.
INNOVATION: Unprecedented ability to image oxytocin at synaptic levels; Readily deployable technology for neurobiology community; Advance understanding of the interplay between social behaviors and ASD.

Functional activities of plasma from autism on blood-brain barrier; Comparison with healthy controls and PANS

PROJECT PI:  Ayan Mondal PhD, Stanford University
PROJECT AIMS: Characterization of unique autism subset of patients experiencing acute behavioral deteriorations resembling Pediatric Acute Neuropsychiatric Syndrome (PANS) and investigate the impact of plasma from these patients on the Blood Brain Barrier function.
INNOVATION: Determine BBB dysfunction mechanisms, potential biomarkers and therapeutic targets using sophisticated BBB models for accurate assessments. Future directions include identifying biomarkers, exploring advanced Emulate brain-on-a-chip technology for accurate representation of BBB phenotypes and develop therapeutics to reduce neuroinflammation during acute deterioration in autism patients.

Identification of the neuronal ensemble activity as a biomarker for the evaluation of treatment strategies

PROJECT PI:  Maltesh Kambali PhD, University of Illinois
PROJECT AIMS: Understand the behavioral features in autism linked to the prefrontal and hippocampal activities in the brain, the NDD-relevant impairments in cognitive functions and social interactions; Investigate the association between rare copy number variations (CNVs) and Neurodevelopmental disorders
Evaluate repurposed FDA-approved drugs bezafibrate (mitochondrial biogenesis activator) and metformin (FDA-approved for diabetes) as potential treatments.
INNOVATION: Potential to identify biomarkers for Blood Brain Barrier dysfunction for\ therapeutic approaches that will in turn lead to clinical trials

BRAIN Research Awards 2022

The gut-brain axis in ASD presenting with gut inflammation

PROJECT PI: John-Paul Yu MD PhD, Assistant Professor, in Radiology (Neuroradiology), Psychiatry, and Biomedical Engineering at University of Wisconsin–Madison
PROJECT FOCUS: To identify how altered gut microbiome in IBD leads to increased risk for ASD by developing a novel human pediatric ASD/IBD microbiome model in mice.
PROJECT AIMS: We will identify altered gut-brain-behavior development in our model and directly test the ability of replacing altered microbiota-derived metabolites to rescue brain and behavior impairments relevant to ASD. Our work will provide the first test of human ASD/IBD microbiota to produce ASD-relevant brain and behavior phenotypes through altered metabolite and brain immune cell signaling. Lastly, we will employ state-of-the-art quantitative multi-compartment diffusion weighted imaging to develop a neuroimaging biomarker for both diagnostic accuracy and to track a microbiota-based therapeutic response.

CRE/FLP system in brain organoids as a tool for illuminating pathology mechanisms

PROJECT PI: Christopher Patzke PhD, John M. and Mary Jo Boler Assistant Professor, University of Notre Dame
PROJECT FOCUS: CRE/FLP system in brain organoids as a tool for acute mutation targeting and dissecting of autism disease pathology mechanism
PROJECT AIMS: This project has two goals: One goal is to study different types of human synapses in in vitro systems that come as close as possible to human brain in vivo situations. Current brain organoid systems are still very far away from being representative for any non-embryonic nervous system. The second goal is to establish a culture system that can be entirely and acutely switched from diseased to healthy genotype and vice versa. Advanced culture techniques paired with genetic engineering will be molecular tricks helping us to understand specific brain regions at any given time in development, in any given medical condition.

Evaluating clinical measures and and biomarkers in behavioural regression in ASD

PROJECT PI: Jennifer Frankovich MD MS, Clinical Professor, Dept of Pediatrics, Stanford University/Lucile Packard Children’s Hospital
PROJECT FOCUS: Illuminating biological underpinnings in behavioural regression in ASD compared to healthy controls and Pediatric Acute-onset Neuropsychiatric Syndrome (PANS).
PROJECT AIMS: Using our longitudinal clinical database and biobank, we aim to describe the clinical and immunological course of patients with autism (n=18) followed in our IBH Clinic through different states (flare, improved, chronic) and compare these data to findings in patients with PANS and to hospital/clinic controls. Immune studies will include monocyte profiles, single-cell RNA sequencing, the effects of plasma from autism patients on healthy monocytes, and blood-brain endothelial cells. Since these experiments will use only a fraction of the stored samples, the remaining will be reserved for cytokine/proteomic profiles, ATAC-seq, and functional assays, so that all these data can be correlated with the clinical data for informing biomarker discovery and clinical trials.

Neuroinflammation and brain development in Autism Spectrum Disorder

PROJECT PI: Paul Ashwood PhD, Associate Professor of immunology at the MIND Institute at the University of California Davis
PROJECT FOCUS: Mapping brain development in autism and correlating it to inflammatory/immune related disturbances.
PROJECT AIMS: Using RNAscope, immunohistochemistry (IHC) and protein arrays, we will measure inflammatory and immune regulatory cytokines in amygdala specimens from control and ASD individuals. Our overall hypothesis is that altered immune regulation maps to the lifelong trajectory of amygdala development in ASD. We will track cytokine levels in different cells to determine whether immune response correlates with brain growth trajectories. We will focus on the balance between regulatory cytokines such as transforming growth factor beta 1 (TGFb1), interleukin (IL)-10 and IL-35, and inflam-matory cytokines such as IL-1, IL-6, tumor necrosis factor alpha (TNFa) and IL-17. We will also attempt to investigate associations with ASD comorbidities and behavioral severity assessments.

Biomarker discovery for autism detection and treatment

PROJECT PI: Karen Parker PhD, Associate Professor (Research) of Psychiatry & Behavioral Sciences, Stanford University
PROJECT FOCUS: Discovery of clinical biomarkers for improved detection and treatment of autism
PROJECT AIMS: To identify robust protein markers of ASD for novel therapeutic target identification and diagnostic test development using an innovative CSF-first strategy. This project is based on promising preliminary data and is statistically well-powered to detect effects. Our specific aims are two-fold: Aim 1: Use the full power of Olink’s state-of-the-art proteomic platform to identify proteins in banked CSF samples that enable accurate differentiation of children with ASD from unrelated child controls. Aim 2: Assess whether the multiplex CSF protein profile is evident in banked blood samples from children with ASD and unrelated child controls

Bio behavioral study of arousal in nonspeaking children with autism

PROJECT PI: Sarah A. Schoen, PhD, OTR, Director of Research, STAR Institute for Sensory Processing
PROJECT FOCUS: Assess the presence of stress patterns impacting functioning in autism
PROJECT AIMS: The over arching goal of this study is to better understand and treat the unique arousal/ stress responses of non-speaking autistic children. Specifically this project will employ novel tools to assess the presence of stress patterns impacting function as well as determine the role of arousal mechanisms in treatment. We will employ a bio behavioral approach for the examination of arousal, and provide intervention that is informed by these tools. We will measure the effectiveness of a unique short term, intensive intervention that focuses on autonomic, sensory, motor and emotional components of arousal with the ultimate goal of increasing client well-being, sense of agency and autonomy.

Autism spectrum disorder inflammatory subtype and long COVID

PROJECT PI: Harumi Jyonouchi MD, Board Certified Allergist/Immunologist, Saint Peter’s University Hospital, Rutgers-Robert Wood Johnson Medical School
PROJECT FOCUS: Immune mediated inflammation in long COVID patients with or without ASD
PROJECT AIMS: The long-term goal of our research is to determine the underlying mechanisms of immune
mediated inflammation in long COVID patients with or without ASD. The immediate goals of this proposal are to assess how COVID infection triggered long-term changes in the innate immune system affect ASD subjects in comparison with non-ASD long COVID subjects. Completion of this project will help identify 1) key pathological changes in long COVID symptoms in ASD subjects, and 2) how these changes affect clinical features of ASD and assess treatment options in these subjects.

Profiling auditory sensitivity in Autism Spectrum Disorder

PROJECT PI: Adam Naples, PhD, Assistant Professor in the Child Study Center at the Yale School of Medicine
PROJECT FOCUS: Specifying the exact nature of auditory processing disturbances in autism
PROJECT AIMS: To determine whether measures of fluctuations in PD and EEG differ between participants with ASD and NT controls. Second, we will determine how measures of fluctuations in PD and EEG affect the day-to-day
experience of auditory sensitivity.

Defining comorbidities in Autism Spectrum Disorder

PROJECT PI: Chandra Menendez PhD, Postdoctoral Fellow in the Department of Microbiology and Immunology at the University of Oklahoma Health Sciences Center
PROJECT FOCUS: The human translational study will identify biomarkers and advance the identification and treatment of basal ganglia comorbidities in ASD
PROJECT AIMS: To investigate the hypothesis that infections and basal ganglia autoimmunity are risk factors in ASD comorbidities. The project will attempt to determine putative AAb biomarkers (including AAbs that activate the dopamine receptors) and immunophenotypic signatures in ASD and correlate them with ASD comorbidities or immune dysregulation. It will also investigate immunophenotypic signatures in ASD subtypes with and without comorbidities.

BRAIN Research Awards 2021

Using a Humanized Mouse Model And Human Intestinal Tissue to Evaluate the Zonulin Pathway For Personalized Treatment of Autism

PROJECT PI: Dr Alessio Fasano, Professor of Pediatrics & Vice Chair of Research at Mass. General Hospital, Professor of Nutrition at Harvard T.H. Chan School of Public Health.
PROJECT FOCUS: gut permeability/microbiome /immune response interplay in autism
PROJECT AIMS: advance our understanding of ASD pathogenesis and the mechanisms behind increased gut permeability; identify biomarkers to stratify the ASD population in specific subgroups; validate the zonulin inhibitor AT1001 (already showing robust safety profiles in phase 3 human trials with the name Larazotide acetate) as a possible personalized therapeutic strategic treatment, so posing foundations for future clinical trials in ASD.

Evaluation of Transcranial Photobiomodulation in Autism: Double-Blind, Placebo-Controlled, Randomized Study

PROJECT PI: Tolga Atilla Ceranoglu, MD, Assistant Professor in Psychiatry, Harvard Medical School and Mass. General Hospital.
PROJECT FOCUS: transcranial photobiomodulation (t-PBM) is a novel form of neuromodulation that is based on non-retinal exposure to light at specific wavelengths. So far it has yielded very promising early results for the treatment of several neuropsychiatric disorders. Its mechanisms of action include increasing mitochondrial energy production, lowering inflammation and increasing regional blood flow in the brain. The treatment has a low cost, good safety profile, and it is easy to self-administer.
PROJECT AIMS: evaluate efficacy and safety of t-PBM treatment of autism and provide the first step towards FDA approval.

Immunoregulation and Gastrointestinal Issues in Autism Spectrum Disorder

PROJECT PI: Paul Ashwood, PhD, Professor of Microbiology and Immunology, MIND Institute, University of California Davis.
PROJECT FOCUS: understand underlying pathological pathways in gastrointestinal (GI) issues in autism and develop treatment strategies.
PROJECT AIMS: determine the mechanisms of increased innate immune activation. The project will examine the hypothesis is that macrophage dysfunction in autism is due to epigenetic changes that cause increased immune activation, decreased endotoxin/bacterial tolerance, and lead to inflammation. The researchers will aim to identify a panel of biomarkers, or biological signatures, that could be used for patient stratification, development of new therapeutic targets, or to monitor treatment responses.

High-Frequency Oscillation as a Biomarker of Mitochondrial Dysfunction Associated with Epilepsy in Autism

PROJECT PI: Richard Frye, MD PhD, Pediatric Neurologist, Phoenix Children’s Hospital.
PROJECT FOCUS: studying mitochondrial dysfunction as a cause of epilepsy in individuals with and without autism as well as the correlation with these areas of mitochondrial dysfunction with high frequency oscillations (HFO) that can be recorded non-invasively as a biomarker of mitochondrial associated epilepsy. Individuals with autism are at an increased risk for epilepsy and seizures. Epilepsy is the leading cause of death in autism.
PROJECT AIMS: deepen our understanding of the biological mechanisms that underlie epilepsy, including treatment-resistant epilepsy, and to open a path to novel biomarkers, assessment methods and treatments.

Autism with Neurodevelopmental Regression Associated Mitochondrial Dysfunction: Further Development of In Vitro Models and Pathways to Treatment

PROJECT PI: Richard Frye, MD PhD, Pediatric Neurologist, Phoenix Children’s Hospital.
PROJECT FOCUS: mitochondrial dysfunction in regressive autism. About one-third of children with autism spectrum disorder demonstrate a major neurodevelopmental regression – loss of previously acquired language and social interaction skills, as well as emergence of restricted and repetitive behaviors.
PROJECT AIMS: The overall goal of this study is to develop better treatments for children with autism. In order to reach this aim, the investigators will further characterize mitochondrial dysfunction in their in vitro models and test agents that potentially modulate mitochondrial function in order to discover novel targeted treatments for this subset of autism.

Early Biomarkers in Autism Spectrum Disorders: Birth Folate Autoantibody Receptor Status

PROJECT PI: Harris Huberman, MD, Pediatrician, SUNY Downstate Medical Center.
PROJECT FOCUS: examine the role of folate receptor auto-antibodies (FRAA) in the development and manifestations of autism. Folate in the central nervous system plays a role in a myriad of metabolic, genomic and brain architectonic processes. FRAAs have been clearly correlated with autism and appear to constitute a significant contributing factor.
PROJECT AIMS: improve understanding of central folate deficiency on infant development; improve understanding how measures of brain activity and social behavior correlate with FRAA status, and later ASD diagnosis; improve early identification of ASD and lay the groundwork for future treatment interventions of high-risk infants with leucovorin.

Biomarkers of Innate Immune Memory in Autism Spectrum Disorders

PROJECT PI: Harumi Jyonouchi, MD, Board-certified Allergist/Immunologist, Saint Peter’s Hospital, Professor of Pediatrics at Rutgers Robert Wood Johnson Medical School.
PROJECT FOCUS: immune mediated inflammation that affects not only the brain but also other organs has been implicated in many of the observed clinical features in autism. However, inflammation in autism appears not associated with specific environmental factors, but appears to be driven by innate immune memory (IIM) responses that are generated by metabolic and epigenetic changes triggered by an initial stimulus.
PROJECT AIMS: determine the underlying mechanisms of immune mediated inflammation and their effects on the development and progress of autism.

Connecting Rare Genetic Mutations in Autism to Common Biological Pathways

PROJECT PI: Joseph D. Buxbaum, PhD, Professor of Psychiatry, Genetics and Neuroscience, Icahn School of Medicine at Mount Sinai. Director of the Seaver Autism Center.
PROJECT FOCUS: study the function of many autism-linked genes simultaneously, in order to identifying a smaller number of common pathways shared by those genes. Apply a computational drug repurposing approach to search for already existing compounds that would be predicted to revert altered ASD pathways to a healthy state.
PROJECT AIMS: improve our understand of how changes in nerve and brain development lead to autism disorder in order to develop effective new treatments for ASD we need to

Using AI & Machine Learning to Develop Metabolic Biomarkers in Autism Spectrum Disorders

PROJECT PI: Dr. Juergen Hahn, Professor & Department Head, Biomedical Engineering, Rensselaer Polytechnic Institute.
PROJECT FOCUS: analyzing measurements of metabolite concentrations of the folate-dependent one-carbon metabolism and transsulfuration pathways to distinguish children diagnosed with autism from their typically-developing peers.
PROJECT AIMS: replicate the results on a new, large cohort of children using state-of-the-art technologies appropriate for a commercial lab test; develop the foundation for autism biomarkers to be used for diagnosing autism and for assessing efficacy of treatments.

Study the Pathological Status in Gastrointestinal Tissue in GI Symptomatic Children with and without Autism

PROJECT PI: Stephen Walker, PhD, Professor of Regenerative Medicine, Wake Forest School of Medicine, and Arthur Krigsman MD, Board-certified Pediatric Gastroenterologist
PROJECT FOCUS: significant molecular pathology is often lurking in the normal or near normal light microscopic images of intestinal biopsy tissue of GI symptomatic children with ASD.
PROJECT AIMS: replicate the existing findings in order to provide support for a change in prevailing clinical practice of the evaluation of GI symptomatic ASD children to include a more thorough evaluation of biopsy tissue when the presenting symptoms are suggestive of an inflammatory phenotype.

MEF2C Haploinsufficiency Drug Discovery - Potential to Improve ASD Phenotypes

PROJECT PI: Stuart Lipton, MD, PhD, Professor of Molecular Medicine, Scripps Research.
PROJECT FOCUS: partial loss of MEF2C activity is known to lead to ASD, intellectual disability, epilepsy and other neurological symptoms. Since MEF2C is a major regulator of the expression of various other proteins known to be involved in various forms of ASD, such a drug may be an effective treatment for a wide variety of ASDs.
PROJECT AIMS: optimize lead compounds discovered by Lipton’s lab in order to develop drug candidates to treat a specific type of autism by increasing the activity of MEF2C neuronal transcription factor.

Pharmacologically Remediable Traits of ASD in a Mouse Model Implicate a Serotonin – Neuroinflammatory Connection

PROJECT PI: Randy Blakely, PhD, Professor of Biomedical Science, Florida Atlantic University.

BRAIN Research Awards 2020

Characterizing Altered Innate Immune Memory in Autism & Exploring Select MicroRNAs as Biomarkers

PROJECT PI: Harumi Jyonouchi, MD, Board-certified Allergist/Immunologist, Saint Peter’s Hospital. Professor of Pediatrics, Rutgers Robert Wood Johnson Medical School.

Understanding Differences in Intestinal Epithelium in Autism and Controls & Constructing a Biobank of Intestinal Organoids

PROJECT PI: Paul Ashwood, PhD, Professor of Microbiology and Immunology at the MIND Institute, University of California Davis.

Microbiota Transfer Therapy for Individuals with Autism Spectrum Disorder, with Extensive Testing

PROJECT PI: James Adams, PhD, President’s Professor, Arizona State University.

Gene-Microbiome Interactions in ASD: the Effects on Neurotransmitter Release, Immune Responses & GI Function

PROJECT PI: Sarkis Mazmanian PhD, Luis & Nelly Soux Professor of Microbiology in the Division of Biology & Biological Engineering, Caltech.

Investigating Chemical Exposures and Crosstalk between the Metabolome and Exposome in Autism

PROJECT PI: Robert Naviaux, MD, PhD, UC San Diego

Explore if Treatments Targeting Mitochondrial Function in Autism Will Improve the Core Symptoms

PROJECT PI: Richard Frye, MD PhD, Pediatric Neurologist, Phoenix Children’s Hospital.


Establish a Rat Model of ASD to Study Genetic & Epigenetic Changes and Associated Behavioural Deficits

PROJECT PI: Edward Quadros, PhD, SUNY Downstate.

Symptom Improvement ASD Children Following Treatment for their Chronic GI Inflammation

PROJECT PI: Arthur Krigsman, MD, Board-certified Pediatric Gastroenterologist.

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