Q: What prompted the creation of Autism Speaks?
Autism Speaks was formed in 2005 by Suzanne and Bob Wright, the grandparents of a child with autism. The Wrights had a vision to create a unified voice for the millions of families affected by autism spectrum disorders (ASD). Within a short period time after launching Autism Speaks, their vision was realized as it successfully merged together each of the key national autism foundations at that time (the National Alliance for Autism Research, Cure Autism Now, and the Autism Coalition for Research and Education). In the eight years since, Autism Speaks has grown to become the world's leading autism science and advocacy organization, dedicated to funding research into the causes, prevention, treatments, and a cure for autism; increasing awareness of autism spectrum disorders; and advocating for the needs of individuals with autism and their families. Autism Speaks has funded $195 million in cutting-edge research and has developed a robust portfolio of essential resources for families. Each year, Walk Now for Autism Speaks events are held in more than 100 cities across North America, and on the global front, Autism Speaks has established partnerships and related activities in more than 40 countries on five continents to foster international research, services, and awareness. On April 2 each year, Autism Speaks celebrates the United Nations-sanctioned World Autism Awareness Day and kicks off autism awareness month with our "Light It Up Blue" campaign, a signature global initiative aimed squarely at increasing awareness internationally.
Q: What were the challenges in autism research that you felt a patient-driven philanthropy could uniquely help address?
Over the past 10 years, enabled in part by funding from Autism Speaks, the state of the science around ASD has matured to a point where the field is in a genuine position to begin converting scientific breakthroughs, along with accompanying innovations in technology, into products with the potential of addressing the wide range of unmet needs (healthcare, educational, and daily living) the ASD community struggles with every day. Unfortunately, the translational research process that develops and delivers these products faces numerous challenges. Patient-driven philanthropy organizations such as Autism Speaks are in a unique position to address these. Some examples that our foundation is actively focused on include:
- Clarifying the regulatory pathway(s) for medical product development: From a regulatory perspective the ASD space represents a largely unprecedented area of product development. From the unavailability of valid outcome measures to general challenges with pediatric development, sponsors and other developers are forced to navigate and pave new pathways to regulatory approval. This also has an unfortunate, and often negative, impact on the perception of risk among investors whose capital is required to support these endeavors. Clarifying regulatory paths and achieving new regulatory precedents will fundamentally change the landscape of translation ASD. In addition to our general efforts to help improve understanding of the unmet needs of ASD, the clinical benefits, and risks of various medical products under development, we have been specifically active in leading consensus-building workgroups that are delivering specific recommendations to the field (including the FDA) on the status of outcome measures for use in supporting medical product development for ASD.
- Increasing access to well-phenotyped clinical biosamples: Repositories of biological samples, from cases with associated high-quality clinical data, are an essential strategic resource for any research field focused on translation. With examples such as our repository of clinical and genetic information (AGRE) and our brain bank (ATP), Autism Speaks has seen excellent return on investment in terms of impacting the research landscape for ASD.
- Improving community participation in research through outreach: Whether serving as a donor of biological samples or participating in a clinical trial for a new experimental medicine, community participation in research is a critical and often rate-limiting factor to the success of translation. Limited access to community participation can also represent a key challenge for developers. Patient groups like Autism Speaks are in a unique position to address this challenge by building awareness among the community on the value and impact their participation can have on research.
Q: Clearly there is still much about the basic science of autism that is yet undiscovered, but Autism Speaks seems to have always been interested in laying the groundwork for future stages of research and development. Can you tell us a bit about your strategies to support translational research and therapy development in parallel with basic research?
The ultimate goal for translational research at Autism Speaks is fairly straightforward: to ensure that scientific breakthroughs and innovations in technology, with potential to transform outcomes for individuals with autism, are developed into products and brought to market expeditiously. Accomplishing these translational goals, however, requires an appreciation for the complexities and challenges of working across the diverse partner landscape needed to progress product concepts through the different stages of research and development. Strategies to support translation must work to create engagement from partners working across the entire value chain, and sustain their focus on a common endgame amid often competing incentives, measures of success/progress, and sources of funding. At Autism Speaks, we know that we must work beyond the science to deliver the science, and from a strategic perspective we recognize that the "business" of translation is as important as the science that originally puts it in motion. Some examples of how we are tackling this element of supporting translation are:
- Innovating funding models: We have seen a need to innovate our funding models beyond the traditional, academia-centric, investigator-initiated award mechanisms. Although these are tried and true vehicles for funding basic research, they may not be fit-for-purpose in supporting partnerships with the for-profit sector (e.g., biotech, pharma). In response, Autism Speaks recently created a nonprofit venture philanthropy affiliate called Delivering Scientific Innovation for Autism (or DELSIA). DELSIA enables the science mission of Autism Speaks through investments in entrepreneurs and companies – entities that have a common focus on developing commercial products and addressing the unmet needs of the autism community in the areas of healthcare, education, and daily living.
- Stimulating capital investment in the autism space: From an autism perspective, the so-called translational research "valley of death" is real, and the ability of scientific innovations to move forward through development has been hampered by insufficient capital investment. In parallel to our general de-risking activities around research and regulatory pathways, we have been developing strategies to increase engagement of the investment community, helping to introduce the value proposition for new product/business development, and provide venues to showcase and connect developers of technologies with the professional investors. From a strategic point of view, we see investment conferences such as the one we just hosted in February 2013 being just as critical to moving the ball forward as scientific conferences.
Q: What do you consider the organization's greatest accomplishments to date?
Science: Since the establishment of Autism Speaks, the foundation has provided more than $195 million in funding to support research through traditional grant mechanisms, fellowships, and staff-directed projects. These awards have helped produce most important published findings in the autism field. We are also enormously pleased with the accomplishments of our portfolio of programs that are considered among the autism field's most valuable and strategic resources. Examples of these include:
- The Autism Genetic Resource Exchange (AGRE): Established in 1997 and operated by Autism Speaks , AGRE is the largest private, open-access repository of clinical and genetic information dedicated to help autism research in the world. Our current understanding of the genetic architecture underlying risk for autism spectrum disorders would not have been possible without the resources available through AGRE.
- The Autism Speaks Treatment Network (ASTN): A collaborative national network of 17 medical centers dedicated to providing families with state-of-the-art, multidisciplinary care. The ASTN was established to provide a place for families to go for high-quality, coordinated medical care for children and adolescents with autism and associated conditions.
- The Global Autism Public Health initiative, which seeks to facilitate the development of systematic and sustainable solutions for enhancing global autism awareness, research, training, and service delivery.
Advocacy: In the area of advocacy, Autism Speaks is pleased to have played a critical role in helping to secure $1.8 billion in federal funding for autism research, treatment, and services through the passage of the Combating Autism Acts of 2006 and 2001, the American Recovery and Reinvestment Act, and the Department of Defense Congressionally Directed Medical Research Program. We also take great pride in our advocacy accomplishments in the area of Autism Insurance Reform, which has helped end marketplace discrimination on the basis of an autism diagnosis. Now 32 states (representing 75 percent of the U.S. population) have enacted laws requiring private insurance health plans to cover the diagnosis and treatment of autism spectrum disorders.
Family Services: Beyond science and advocacy, we are proud of our accomplishments over the years in the area of family services, where we have developed a number of exceptional tool kits and other resources that help guide individuals with autism and their families throughout their lifespan. These include a 100 Day Kit for newly diagnosed families, School Community Tool Kit, Transition Tool Kit, Family Support Tool Kits, and online Resource Guide. We also co-founded Advancing Futures for Adults with Autism in 2008, a national consortium of organizations working together, led by the vision of individuals with autism and their families, to promote a collaborative spirit and develop both public and private sector support that improve the lives of adults living with autism.
Q: What are your top research goals for the next year, and what will it take to reach those goals?
As we enter into 2013, we have begun to execute the first year of a five-year strategic plan for science that provides a framework that will guide the development of new programs and shape the continued evolution of our current portfolio. The primary areas of focus include:
- Identifying risk factors for autism that can lead to prevention and improved diagnosis and treatment. Leveraging genomic and bioinformatics resources to accelerate the discovery of genetic and environmental risk factors, diagnostic methods, and predictive and early efficacy biomarkers.
- Promoting and facilitating the development of safe, effective interventions and medicines. We are working to facilitate the development of therapeutics that address both the core features of ASD and the range of associated comorbid psychiatric, neurological, and somatic symptoms.
- Enhancing the quality of, and access to, comprehensive healthcare for individuals with ASD. Much of our activity in this area is focused on improving the capabilities and capacity of our Autism Speaks Treatment Network (ASTN), which is a network of 17 centers across North America dedicated to improving medical care for children and adolescents with autism by offering comprehensive diagnosis, treatment, care, and counseling.
- Improve the health and outcomes of adults with autism from a lifetime perspective. We are looking to identify critical periods during which various interventions, supports, and services are crucial for best outcomes with a special focus on factors that promote positive outcomes in adults.
- Reducing the age of early detection and improving access to behavioral interventions. Towards this goal, we have just launched our Early Access to Care program, an initiative that seeks to reduce the average age of diagnosis and increase access to high-quality early intervention for all children on the autism spectrum.