Centered Around Autism

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Courtesy of OC Kids

UC Irvine recently received $14.8 million in funding to create a center dedicated to finding a treatment for autism.

With $14.8 million of financial support from private donors of the William & Nancy Thompson Family Foundation and the Children & Families Commission of Orange County (CFCOC), UC Irvine hopes to bring a viable treatment to children in the U.S. afflicted by autism, a condition that affects one out of every 88 children.

UCI recently announced at a launch in mid-December the creation of the Center for Autism & Neurodevelopmental Disorders of Southern California, which will combine both clinical and research aspects of treating the disorder.

“This new center is really amazing in what it can accomplish for families living with ASD and researchers, clinicians and educators working in this area,” Gillian Hayes, the director of technology research for the center, said. “This is one of only a handful of places that supports integrated care across a variety of disorders from birth through adulthood.”

Originally intended to be one entity, the center will exist as a two-pronged force with both clinical aspects and translational research approaches to try and remedy autism — from the genetic level all the way to the behavioral components.

“The vast majority of the funds go to support the clinical enterprise — taking care of the kids. But, a very substantial portion goes to support the research done here at UCI,” Dr. J. Jay Gargus, the lead director of research for the center, said.

“Our whole goal in the research center CART (Center for Autism Research and Treatment) is drug discovery for autism,” Gargus said. “It’s meant to take very basic research that is being done here at UCI and take it out to make a small-molecule therapeutic that we can put into kids.”

Bringing together over 40 faculty researchers from across multiple disciplines at UCI, Gargus has created a team under a common goal: making a platform to treat autism.

“The excellent professional staff, clinicians and researchers will all work together cohesively to support quality health and educational outcomes for all the patients and families they serve,” Hayes said.

With big pharmaceutical companies shutting down their autism research branches in recent years due to failures of translating research from mice to humans, Gargus’ team of researchers remains one of few seeking a therapeutic method to treat those with autism.

Looking at the disorder from the most basal level, the genes, and carefully taking the treatments step-by-step, Gargus hopes to be able to not only create ideal models, but also take those models and translate them into therapeutics for humans.

“We are going to be looking at all of these molecular events along the way because we want to see a very secure signal that we are fixing the molecular region that many types of autism have in common,” Gargus said. “We’re going to be tightly focused on function, not just jumping directly to the behavior — we want to know that we’re really fixing what we think is broken.”

The researchers have developed a small core of 200 research subjects that they have studied extensively in the preliminary stages, with rare forms of autism caused by lesions of one or two genes. However, their hopes of understanding the full complexity of the disorder will only come from further studying the patients that aren’t “ideal.”

“What we want to do is get our system working on these well-understood patients, then start bringing in the typical patients — they’re going to be genetically more complicated,” Gargus said. “In order to bring in those patients, we’re going to be genotyping their entire genome sequence. It’s a phenomenal resource.”

And with the research component being successfully supported, this means great prospective treatments for all of the patients that the center has. The center is currently determined to take necessary steps to integrate the patients from the center into the pipeline of research trials that will be underway in the near future — testing safe compounds similar to vitamins that researchers already know are safe for human subjects.

“We want to open it up,” Gargus said. “We want to be one of the sites that when we have a clinical trial we’re going to be able to have that available to the people here — and that’s really going to be our promise.”