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Autism & Developmental Disorders Inpatient Research Collaborative (ADDIRC), Spring Harbor Hospital, Westbrook, ME
2013 - 2015

Principal Investigator(s): Matthew Siegel, M.D., Maine Medical Center

Phenotyping of the Severely Affected Autism Population: Developing a Research Platform for the Study and Treatment of the Severely Affected Autism Population (Co-funded with the Simons Foundation)

Although rigorously collected phenotypic and biological data have contributed greatly to ASD research, adequate data from severely affected individuals are lacking. Barriers to the study of the severely affected ASD population include challenges in their recruitment and participation in outpatient research studies, the limited contact of most investigators with this population, and the relative lack of validated measures for characterizing these individuals.

The Autism and Developmental Disorders Inpatient Research Collaborative (ADDIRC) is dedicated to becoming a unique and singular research platform for advancing the clinical assessment and treatment of patients with severe ASD. Each year, over 1000 children and adolescents with ASD and serious behavioral disturbance are admitted to the nine specialized psychiatric hospital units that comprise the ADDIRC. These hospital units are staffed by multi-disciplinary teams of highly skilled clinicians and investigators with unique expertise in assessing and caring for these patients. The ADDIRC patient population is heavily weighted toward individuals with severe ASD.

The ADDIRC is engaged in a two-year project to establish multi-site data collection procedures and to test these procedures in a prospective study of its inpatient ASD population. The project team, including clinical and research scientists from each ADDIRC site and scientific and administrative advisory groups, has expertise in the assessment of severe ASD, the collection and analysis of phenotypic and genetic data and the administration of collaborative research. The immediate goal of the network is to develop a comprehensive registry for the collection of clinical and eventually biological data on severely affected children and adolescents with ASD in order to systematically develop effective interventions for this population. The network will collect detailed data on the severely affected population, including the dimensions of intelligence, communication ability, emotional regulation and self-injurious behavior, and examine the relationships among these critical factors.

Autism & Developmental Disorders Inpatient Research Collaborative (ADDIRC)

Bradley Hospital/Brown University, Providence RI

Principal Investigator: Lindsay Oberman, Ph.D., Brown University

Career Development Award

This grant provides support for Dr. Lindsay Oberman in a translational research project that will extend and bridge two independent lines of research, both previously funded by the NLMFF. Specifically, with funding from NLMFF as well as NIH and Harvard Catalyst, Dr. Alvaro Pascual-Leone and Dr. Oberman have developed methods to noninvasively measure experience-dependent cortical plasticity both in healthy controls and patients with idiopathic ASD and Fragile X syndrome. Using noninvasive repetitive transcranial magnetic stimulation (rTMS), Drs. Oberman and Pascual-Leone have shown that patients with idiopathic ASD show an exaggerated LTD-like suppression of cortical excitability following a short train of rTMS while those with Fragile X (without ASD) syndrome show a complete lack of LTD-like suppression in response to the same rTMS protocol.

Under a separate line of research, also previously funded by the NLMFF, Dr. Matthew Anderson developed a mouse model of ASD based on triplication of the UBE3a gene (the genetic mutation that causes idic15 in humans) that reconstitutes correlates of the three core behavioral deficits that define ASD. Furthermore, they have developed a model mechanism where they propose that social deficits in individuals with idic15 may be a consequence of excessive experience-dependent social homeostasis.

Independently, these two lines of research have both contributed to our understanding of the underlying pathophysiology of the behavioral deficits that characterize ASD. A complete understanding, however, requires the direct translation of insights that we gain from basic science to applications that have direct impact for patients with the disorder. With this focus, this project aims to create a multi-disciplinary collaboration between the Anderson and Pascual-Leone lab with Dr. Oberman as the catalyst of this translational bridge. Thus, the aim is to develop novel assays, based on the previous work in the Anderson and Pascual-Leone lab, to evaluate neurological and behavioral phenotypes in human patients with a specific syndromic form of ASD, idic15.

Institute on Communication and Inclusion, Syracuse University, Syracuse, NY
2013 – 2015

Principal Investigator: Christine Ashby, Ph.D.

Integration of iPads and Other AAC to Improve Communication for Individuals with Autism

Dr. Ashby’s research team aims to understand the potential of the iPad and other mobile technologies in supporting communication and inclusion of individuals with autism. What applications are most useful for individuals who do not speak or whose speech is highly limited? How can the iPad help individuals with autism develop greater independence, improve their motor planning, or develop verbal speech? Also, while the iPad has nearly unlimited potential, Dr. Ashby’s research team also wants to understand how it can be meaningfully integrated in school and community settings along with other communication strategies to increase meaningful access to academic, work, and social experiences. Technology alone is not sufficient; training and ongoing support is necessary to ensure that use of the technology enhances communicative interactions and educational access. Many schools and agencies are purchasing iPads with no plan for meaningful integration and no plan for how this new technology fits into a larger total communication approach.

The goal of this project is to enhance our understanding of the potential for iPads and other mobile AAC devices to support communication. Through this grant, Dr. Ashby’s research team will explore, evaluate, and organize applications that are most useful in helping non-speaking individuals with autism develop skills related to typed communication and achieving independent communication. The grant will also support the development of a pilot app, a multifaceted assessment tool that will aid in determining candidacy for facilitated communication training, current pointing skills and literacy levels. Finally, this project will focus specifically on the use of the iPad for helping individuals with autism develop greater physical independence when typing to communicate.

Institute on Communication and Inclusion

Massachusetts Advocates for Children, Boston, MA
2002, 2003, 2004, 2005, 2006, 2007, 2008, 2009, 2010, 2011, 2012, 2013

Establishment and Support of the Autism Special Education Legal Support Center

The goal of this project is to provide training, technical assistance, and advocacy services necessary to ensure that children with autism receive equal educational opportunities. Goals include: Providing parents with information about state-of-the-art services and programs available to meet individual needs of students with disabilities; Insuring that children with autism receive special education services necessary to reach their potential in areas impacted by their disability; Increasing public awareness and understanding of the potential and competency of individuals with autism, targeting policy makers, media, educators, service providers, as well as the general public. The Autism Special Education Legal Support Center will accomplish these goals by: providing community-based workshops for parents, educators, and medical professionals regarding legal rights and range of service options available for children with autism; providing a hotline to give legal and technical assistance to families of children with autism; training attorneys to increase representation of low-income students with autism to ensure that children receive legally mandated special education services; and providing information to the media, the legislature, and other policy makers regarding changes necessary to ensure children with autism receive services that reflect their potential.

Click here to read the NLMFF Interview with Massachusetts Advocates for Children

Massachusetts Advocates for Children

Massachusetts Institute of Technology, Cambridge, MA
2013 – 2016

Principal Investigator(s):
Ann Graybiel, Ph.D., MIT
Bernardo Sabatini, Ph.D., Harvard Medical School
Guoping Feng, Ph.D., MIT

Synaptic and Behavioral Functions of Striatal Projection Neurons in Autism and Autism Spectrum Disorders

The grant supports complementary activities in the laboratories of Professor Guoping Feng and Professor Ann M. Graybiel at MIT’s McGovern Institute for Brain Research and Professor Bernardo Sabatini at Harvard Medical School. It involves both a fellowship program and a research program.

The three-year fellowship program will enable the principal investigators to train and mentor one postdoctoral fellow in their respective laboratories to work on autism research. The primary goal of this postdoctoral training program is to provide postdoctoral fellows with intellectual challenges and a broad range of advanced research techniques that will shape their future as independent scientists and leaders in the field of autism research. The fellows will be given an opportunity to contribute to cutting edge research into understanding the neural mechanisms underlying autism spectrum disorders. During their fellowships, the postdoctoral fellows will have the opportunity to use genetic, biochemical, electrophysiological and behavioral approaches to dissect molecular, synaptic, and circuitry mechanisms of ASDs using animal models. These projects will provide them with extraordinary training in four areas that will be key to achieving their long-term career goals. First, it will give them solid training in molecular, cellular, and biochemical approaches to studying synaptic development. Second, it will provide them with excellent training in using genetic approaches in mice to study synaptic function and behavior. Third, it will provide training in using electrophysiological approaches to study synaptic and circuitry function and dysfunction. Fourth, it will provide them with training in animal behavioral tests.

Professors Feng and Graybiel will embark on a concentrated research effort aimed at understanding how the synaptic and behavioral functions of the striatum are critical to autism and autism spectrum disorders (ASD). The focus of their research will be the medium spiny neurons of the striatum (known in the field as MSNs), because these neurons and the circuits in which they are embedded are now suspected to be key brain components affected in autism and ASD. Professor Bernardo Sabatini’s laboratory will focus on developmental and dynamical control of striatal circuitry by dopaminergic inputs.

Bernardo Sabatini Lab

Graybiel Laboratory

Guoping Feng


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