Research

The Center for Autism Research and Translation at UC Irvine aims to confront traditional roadblocks to the development of autism therapeutics, which has the potential to profoundly change the way treatment strategies are evaluated. 

Autism research is at the cusp of being able to benefit from recent discoveries in a number of neurological disorders, such as seizures, where some individuals are genetically predisposed to the disease. In others, genes and environmental stressors — chemical, physiological and psychological — interact to provoke the condition. 

In autism, mutations in single genes cluster in biological pathways — the likely final common pathway — that involved deficits in brain neuronal synaptic communication. Defects result from malfunctions influenced by genes and environmental stresses acting through genes. Consequently it is now possible to study how a number of insults from the environment can further influence the path toward disease. 

Drugs that normalize a shared common pathway would be powerful tools to design and carry out high-throughput cellular screens to test for correction of this molecular defect. Successful screen assays are now rendered "bioassay diagnostics," a dependable molecular surrogate for the complex behavioral abnormalities of the disease that can themselves become clinically useful.

Drugs that can normalize the bioassay screen of an autism sample become promising clinical trial candidates for normalizing the brains and behaviors of those with autism. We thus define a target for therapy in autism, and use multiple and multidisciplinary teams assembled in CART from faculty already working in related areas, to identify the consistent abnormalities, and consistently correct the defects.

Additionally, CART has front-loaded this discovery pipeline with:

• Three distinct UC Irvine-patented novel molecular scaffolds targeting distinct classes of receptors implicated in autism
• Two drugs known to be safe in humans after they are re-purposed from successful Phase 1 trials

CART also launches with a full pipeline because of ongoing UC Irvine clinical trials in models of autism (Angelman, Prader-Willi and Rett) as one of three sites in an ongoing NIH-supported rare diseases consortium.