CART Symposium

On Tuesday, November 7th, the UCI Center for Autism Research and Translation (CART) gathered the community to the Cove to discuss current autism spectrum disorder (ASD) research practices and findings. CART’s mission is to, “abolish current and future cases of autism, a severe neurodevelopmental disorder, through discovery, implementation, education and advocacy of novel care, so no child is lost to autism.” The symposium provided a forum for meaningful dialogues between families faced with ASD, subject matter experts, and key influencers.

Dr. J. Jay Gargus, Director of CART, started the symposium with an overview of the current ASD environment. Discussing the limitations of current treatments for ASD, Dr. Gargus mentioned existing therapeutics are predominantly behavioral, and current drugs only address symptoms rather than the core deficits in ASD. Challenges in understanding autism also reside in its heterogeneity, with over 800 different genes accounted for so far in ASD’s genetic architecture. Studies in this area also vary in their mechanisms and definitions, making it difficult to standardize data and findings from the research.

To provide context to CART’s efforts, a parent shared a personal story about her children who have been participants in UCI’s research studies for several years. “When you go back to your offices and labs and do your research, I pray my family’s story has touched your heart, and you remember how much you make a difference. It gives comforts to families with ASD. The work you do comes from real children with ASD whom each have their own story,” said the parent.

Highlights from each session of the day included: a talk by Donald R. Blake - Professor, Department of Chemistry - Analysis of carbon monoxide (CO), carbon dioxide (CO2), methane, and volatile organic compounds (VOCs) in the exhaled breath of subject affected by autism, in which Blake’s group used sensitive atmospheric instruments to measure the breath of autistic children and found different signatures than appeared in the control group. This method could serve as a potential diagnostic measure.

Kelvin Gee in his talk, Targeting the alpha 7 nicotinic receptors to treat the core and associated symptoms of ASD, reviewed early stage work on AVL 3288, a potential drug for ASD that targets alpha 7 nicotinic receptors as well as GABA receptors. In mice, it improves sociability, reduces repetitive behaviors, and also improves cognitive flexibility and function without affecting locomotor activity. Phase Ib trials are upcoming.

Ramesh Srinivasan, Ph.D., Department of Cognitive Sciences & Dept. of Biomedical Engineering discussed Brain Mapping Technology for Identifying Biomarkers in Autism. Srinivasan’s group is measuring activity in brains of autism-diagnosed children. So far, his group has obtained data in 60% of 48 children in a study group. Srinivasan noted that most brain imaging data of autism published today only involves high-functioning, high IQ students. This may be the first large dataset collected of a more variable population who are both high and low functioning. Srinivasan noted that the EEG-based brain imaging is only diagnostically valuable for children under 13, as children older than 13 are indistinguishable from normal controls.

The researchers are building a low-cost device to record magnetic fields in the brain using Sagnac MEG, a technology first developed to find submarines. This device can also record without touching children, important for children with sensory issues. Their next challenge is to scale up the technology and make it more cost effective.

Dr. Michael Leon Ph.D., professor, Dept. of Neurobiology & Behavior, discussed a program of environmental enrichment. Leon and colleagues conducted two randomized clinical trials on children to see if autism symptoms in children could be helped by enriching their environment. 

Leon reported that sensory enrichment therapy raised IQ compared to results obtained from standard care, significantly decreased of symptoms, reduced sensory symptoms after 6 months, and improved receptive language abilities. 

These sensory enrichment techniques are now commercially available as Mendability (mendability.com), a commercially available internet program used by thousands of people around the world. Parents receive novel exercises to do every two weeks that combine two different types of sensory stimulation simultaneously in two daily 15-30 minute sessions. The researchers have found that compliance with the program improved outcomes in a population of 1000 children (real world users). Mendability was effective for males and females at all ages and results did not depend on symptom severity. 

Leon mentioned that autism generally has co-existing conditions. Children using Mendability showed similar levels of improvement for associated conditions. The effects of sensory enrichment therapy can help a broad range of symptoms. While there are children who do not respond, this can be linked to lack of compliance with using the program. The effects of the program seem to be lasting, however, and in many situations, there is also a dramatic improvement in the level of users’ social interactions.