Autism Speaks Introduces New High Risk-High Impact Initiative

Two Million Dollars Awarded to First Round of Collaborative Research Workgroups

NEW YORK, N.Y. (January 15, 2009) Autism Speaks announced the launch of its novel High Risk-High Impact (HR-HI) research initiative, conceived to support cutting edge research in areas that are under-investigated or which could benefit substantially by bringing in outside experts to accelerate progress. The HR-HI initiative seeks to advance efforts that are unusual or of less certain feasibility and outcome, yet have the potential to change the way autism is understood and treated.

Concurrent with this launch, the Autism Speaks HR-HI initiative has already assembled collaborative research workgroups and awarded two million dollar to five ambitious proposals across a diverse range of areas. These projects will investigate topics including the poorly-understood cognitive abilities of individuals who do not use spoken language; the incidence and biology of metabolic dysfunction; the generation of more rapid and inclusive methods for acquisition of brain imaging data; and the development of novel means for large-scale biomaterial collection.

"The High Risk-High Impact initiative brings fresh ideas and talent to autism research," explains Dr. Geri Dawson, Chief Science Officer, Autism Speaks. “In facilitating opportunities for collaboration, we can hasten the path to potentially life-changing results.”

A HR-HI steering committee, responsible for selecting topic areas and helping guide the vetting process, was comprised of Pat Levitt, Ph.D. (Vanderbilt University), Portia Iversen (co-founder of Cure Autism Now), Sarah Spence, M.D., Ph.D. (National Institute of Mental Health), and Matthew State (Yale University), M.D., Ph.D., and Sophia Colamarino, Ph.D. (Autism Speaks), along with HR-HI staff lead Leanne Chukoskie, Ph.D. and Chief Science Officer, Geraldine Dawson, Ph.D. The HR-HI steering committee initiated its efforts at the beginning of 2008 with focused research meetings and in late 2008 culminated with external peer review of the final proposals leading to funding this first round of workgroups. The goal is to actively form and support closely-collaborating groups of research investigators in areas that have the potential to transform the way we think about autism spectrum disorders (ASD).

To facilitate and monitor these research efforts, the HR-HI staff will remain in close contact with the researchers to ensure that these new investigators and teams have access to the wealth of other scientific resources and the knowledge base provided by Autism Speaks and the entire autism community.

The first area of need identified by the HR-HI Steering Committee addresses the subgroup of individuals with autism who are non-verbal and often referred to as “low-functioning.” Non-verbal individuals are nearly always presumed to have low intellectual capabilities, in part because they have no way of demonstrating their cognitive capacities. Yet some of these individuals have developed the ability to type and communicate their thoughts through alternative means, leading to a startling possibility that many more individuals may be cognitively intact but simply unable to access the ability to communicate. If true, this would have profound implications for their treatment and education, but unfortunately very few researchers are working with this subgroup. Therefore, two of the five HR-HI proposals were designed to focus on developing the means to characterize and, hopefully, treat such non-verbal, presumptively “low-functioning” individuals.

The ability to fully comprehend autism has been hindered in part by our inability to work with individuals with autism when behavioral characteristics or other features impede their compliance. Because of this, most of what we know about autism comes from studying higher-functioning and/or older individuals who more readily participate in study efforts. If we are to move forward to a cure, new technologies must be created that overcome behavioral barriers in order to characterize brain functioning in all types of autism and at all ages. Toward this goal, the third HR-HI grant proposes to demonstrate the ability to use novel computational technologies to achieve two minute fMRI scans to assess “resting-state” functional connectivity in the brains of young children with autism while they are sleeping. Getting a picture of brain function, especially at young ages, is critical to gaining a clearer insight into the brain dysfunction in autism. The team of HR-HI researchers hopes that these pioneering studies will more conclusively test the hypothesis that disordered brain connectivity is a central component to the challenges of autism.

Addressing another of the original priorities of the HR-HI initiative, the fourth grant is based on the need to gain a comprehensive understanding of other body systems in autism, not exclusive to brain function. For instance, although some research studies had suggested that metabolic dysfunction may be involved in autism, this field requires clarity on if and how typical metabolism may be disrupted in autism, especially because it has direct implication for treatment strategies. Therefore, this fourth HR-HI grant catalyzes a new working collaboration between two of the top teams in the world in mitochondrial medicine, enabling them to now turn their attention to autism. The teams have put together an exciting set of experiments that span metabolism, genetics and, most importantly, vetting of new techniques that may be used to non-invasively assess mitochondrial function. Development of non-invasive investigative methods will be essential to getting more people with autism assessed for mitochondrial dysfunction.

Finally, the fifth HR-HI grant has the potential for making tremendous impact on recruiting increased numbers of subjects for autism research, and in turn, hopefully accelerating the pace and progress of research. It is becoming clear from studies in other complex disorders that understanding genetic contributions requires recruitment of not just a few thousand, but tens of thousands of individuals providing DNA samples for genetic analysis. The major hurdle in obtaining large numbers of subjects for autism research is the diagnostic bottleneck – it simply takes too long to get a research-reliable diagnosis of autism to obtain these numbers cost-effectively. This project will aim to leverage Autism Speaks' previous investments in the IAN internet community of families in order to develop an alternate strategy to recruit, validate the diagnosis of, and collect DNA on a large population of individuals as rapidly as possible. The investigators' goal is to recruit 6000 children with autism in a single year. If successful, in one year it would nearly double the number of individual samples already available for analyses of various types.

“To put these complicated projects together, investigators have come from both in and outside of traditional autism research,” explained Dr. Dawson, “and all have shown a passion for their research and a commitment of time and energy for the benefit of people who live with ASD. Abstracts for these unique HR-HI projects can be found here. In addition, each of the efforts will be individually profiled in more detail in the upcoming months as we follow the progress of the HR-HI initiative.

About Autism
Autism is a complex brain disorder that inhibits a person's ability to communicate and develop social relationships, and is often accompanied by behavioral challenges. Autism spectrum disorders are diagnosed in one in 150 children in the United States, affecting four times as many boys as girls. The prevalence of autism has increased tenfold in the last decade. The Centers for Disease Control and Prevention have called autism a national public health crisis whose cause and cure remain unknown.

About Autism Speaks
Autism Speaks is dedicated to increasing awareness of autism spectrum disorders, to funding research into the causes, prevention and treatments for autism, and to advocating for the needs of individuals with autism and their families. It was founded in February 2005 by Suzanne and Bob Wright, the grandparents of a child with autism. Bob Wright is Senior Advisor at Lee Equity Partners and served as vice chairman, General Electric, and chief executive officer of NBC and NBC Universal for more than twenty years. Autism Speaks merged with both the National Alliance for Autism Research (NAAR) and Cure Autism Now (CAN), bringing together the nation's three leading autism advocacy organizations. To learn more about Autism Speaks, please visit www.autismspeaks.org.