Autism Spectrum Disorder Diagnostic/Therapeutic Agent

DESCRIPTION provided by applicant Autism Spectrum Disorder ASD is a significant medical problem ASD affects approximately of the population and extracts enormous emotional and financial costs from the afflicted and their families Despite years of research we understand very little about the molecular triggers of ASD and preventative therapeutics remain elusive Recently maternal immune activation has been gaining acceptance as a likely environmental trigger Indeed a growing body of evidence suggests that specific brain reactive antibodies in women may put their in utero child at risk for ASD To date the researchers working in this area have focused on the pathologic activity of serum or polyclonal antibodies isolated from the mothers of an ASD child Spark Flame Inc S F has licensed intellectual property from the Diamond laboratory at The Feinstein Institute for Medical Research andquot Feinsteinandquot who has recently taken this analysis to a new level by cloning individual brain reactive monoclonal antibodies from mothers of ASD children and demonstrating that when injected intravenously into pregnant mice such monoclonal antibodies lead to abnormal brain development and ASD related behavioral abnormalities in male but not female offspring This gender preference in mice is especially intriguing in view of the fact that ASD overwhelmingly nearly afflicts male children Since approximately of mothers of a child with ASD harbor brain reactive antibodies we are now poised to use these brain reactive monoclonal antibodies to advance a commercial ASD diagnostic and ultimate therapeutic modality that will identify mothers whose unborn child is at risk for this devastating disorder The objective of this application is to achieve a realistic proof of concept milestone in creating an ASD diagnostic therapeutic product We will test our hypothesis and accomplish our objectives of this proposal by pursuing two Specific Aims Identify those antibodies that alter fetal brain development Expand the panel of anti brain monoclonal antibodies obtained from mothers of children with ASD With the successful completion of these Specific Aims we will attract further funding from the Federal or private sector With SBIR Phase funding we will continue focusing on the ASD diagnostic as well as developing a andquot decoy antigenandquot therapeutic approach based on the antibodies that alter brain development Such an ASD preventative therapeutic offers the advantage that it will be non immuno suppressive and can be administered during a circumscribed time window PUBLIC HEALTH RELEVANCE Autism Spectrum Disorder ASD is a complex neurodevelopmental disorder characterized by abnormalities in three areas communication social interaction and stereotypic behavior Currently no ASD diagnostic exists and therapeutic treatments are largely restricted to non medical interventions Herein we describe a research program that seeks to advance a commercial ASD diagnostic and matched therapeutic approach