Project Detail
Interagency Autism Coordinating Committee (IACC) logo
Office of Autism Research Coordination (OARC) logo

TrkB agonist(s), a potential therapy for autism spectrum disorders  

Autism spectrum disorders (ASD) are complex developmental disorders that cause problems with social interaction and communication. In addition to the classical idiopathic autism, Rett syndrome (RTT) and several other genetic disorders including Down's syndrome, fragile X, and mental retardation are frequently associated with autism. Altered regulation of brain derived neurotrophic factor (BDNF) has been found in autistic patients. Similarly, in Rett syndrome (RTT) mouse models, BDNF expression is substantially reduced and genetic elevation of BDNF attenuates behavioral deficits. The application of BDNF recombinant protein in therapeutic intervention has not been successful, because BDNF binds to extracellular cholesterol with high affinity preventing its distribution to different tissues. However, novel small molecule compounds (TrkB agonists) that mimic the function of BDNF in the brain have been recently identified. In this study, investigators will assess whether activation of BDNF signaling through small molecule TrkB agonist(s) can lessen the severity of behavioral deficits in RTT mouse models. In this preclinical mouse study, researchers hope to identify a novel treatment for ASDs. Project Status


Funder National Institutes of Health
Fiscal Year Funding $269,500.00
Current Award Period 2009-2011
Project Number 5R21MH089518-02
Principal Investigator Sun, Yi
Received ARRA Funding? Yes
Strategic Plan Question Question 2: How Can I Understand What Is Happening? (Biology)
Subcategory Molecular Pathways
Strategic Plan Objective Green dot: Objective has greater than or equal to the recommended funding. 2SD. Launch three studies that target improved understanding of the underlying biological pathways of genetic conditions related to autism (e.g. Fragile X, Rett syndrome, tuberous sclerosis complex) and how these conditions inform risk assessment and individualized intervention by 2012. IACC Recommended Budget: $9,000,000 over 5 years.
Federal or Private? Federal
Institution University of California, Los Angeles
State/Country California
Web Link 1 TrkB agonist(s), a potential therapy for autism spectrum disorders (External web link)
Web Link 2 No URL available.
Web Link 3 No URL available.
History/Related Projects TrkB agonist(s), a potential therapy for autism spectrum disorders | $269,500.00 | 2009 | 1R21MH089518-01