Peter BellThe Autism SummitOctober 10, 2008

“Solving the AutismPuzzle through Science”

History of Autism

Age of Hope

1990’s – present1940’s – 1960’s

Age of Denial Age of Defeat

1960’s – 1980’s

The Difference of a DecadeAutism in 1995

• Rare (1 in 2,500)• Poorly understood• Not treatable• Lifelong disability• Nothing you can do• NIH Budget: $5MM

Autism in 2005• Epidemic (1 in 166)• Better understood• Treatable• “Recovery” stories• Early Intervention• NIH Budget:

$90MM+

What Changed?• Parents refused accept status quo

– CAN, NAAR and DAN! founded in 1995– Built a vibrant autism research field– Created new resources (AGRE, ISAAC,

ATP)– Lobbied for more government spending

• Children’s Health Act of 2000• Combating Autism Act of 2006

– Raised critical awareness– Gave families hope

Autism Today• Annual spending: $150-200 million• 1 in 150 prevalence

– 1 in 94 boys• Huge awareness gains

– UN designates World Autism Awareness Day• Private/public partnerships• Emergence of translational research• Lifespan issues – the face of autism is

changing

About Autism Speaks • February, 2005

– Autism Speaks founded by Bob and Suzanne Wright

• August, 2005– Completed merger with the Autism Coalition

for Research and Education (ACRE)• February, 2006

– Completed merger with National Alliance for Autism Research (NAAR)

• February, 2007– Completed merger with Cure Autism Now

(CAN)

Autism Speaks MissionScienceScience

FamilyFamilyServicesServices

AdvocacyAdvocacy

AwarenessAwareness

Science• Research

– Investigator Initiated Grants– Initiatives/Conferences

• Clinical Programs– Research Resources– Treatment Networks– Bioinformatics

• $30 million in 2007, $36 million in 2008

Research Program• Etiology Portfolio (What causes it?)

– Genetics, Environment, Epidemiology• Biology Portfolio (What is it?)

– Neuroscience, Pathology, Molecular Biology• Diagnosis Portfolio (How do we know who has

it?)– Early Identification, Phenotypes, Co-Morbidity

• Treatment Portfolio (How do we make it better?)– Biomedical, Behavioral, Technological

Research Framework

“From Bench to Bedside”

What We Fund• Investigator-Initiated Grants

– Pilot Awards– Basic & Clinical Awards– Training Grants

• Mentor-Based Fellowships (MBF)• Physicians Investigator Beginning Autism Research

(PIBAR) – Treatment Grants

• Pharmacological• Complimentary and Alternative Medicine (CAM)• Special Interventions

– Innovative Technology (ITA)– Environmental Science Awards – New!

What Else We Fund• Initiatives

– Autism Genome Project (AGP)– High Risk/High Impact (HR/HI)– Baby Siblings Research Consortium (BSRC)– International Epidemiology Network (IEN)– Environmental Exposures Subcommittee– Scientific Research Meetings & Conferences

• Summits/Workshops: GI, Immunology, Neuroimaging• Conferences: International Meeting for Autism Research

(IMFAR), Society for Neuroscience

What Else We Fund• Resources and Clinical Programs

– Autism Genetic Resource Exchange (AGRE)– Autism Tissue Program (ATP)– Autism Treatment Network (ATN)– Autism Clinical Trials Network (ACTN)– Interactive Autism Network (IAN)– Internet System for Assessing Autistic Children

(ISAAC)

IACC Strategic Plan• CAA requires the IACC to develop

and annually update a strategic plan for ASD research

• Planning process began in Nov 07• SP draft completed in Aug 08• IWG developing cost estimates and

prioritization• IACC to approve final SP by Nov 08

Strategic Planning Process• Nov 07 IACC approved SP process• Dec 07 SP Workgroup formed• Jan 08 Public input solicited

Scientific workshops convened• Feb 08 First Workgroup meeting held• Apr 08 Second Workgroup meeting held• May 08 Town Hall meeting held• Jun 08 Short- and Long-Term Obj’s

developed• Aug 08 Draft SP issued• Sep 08 Public input on draft SP solicited

Vision and Mission of SP• Vision

– The Strategic Plan will accelerate and inspire research that will profoundly improve the health and well being of every individual on the autism spectrum across the lifespan. The plan will set the standard for public-private coordination and community engagement.

• Mission– The purpose of the Strategic Plan is to focus,

coordinate, and accelerate high quality research and scientific discovery in partnership with stakeholders to answer the urgent questions and needs of individuals on the autism spectrum and their families.

Core Values of Strategic Plan• Sense of Urgency• Spirit of Collaboration• Consumer-Focused• Scientific Excellence• Partnerships in Action• Accountability

Cross-Cutting Themes• Heterogeneity• Early Detection• Lifespan Perspective• Data Sharing• Resources• Public/Private Partnerships

Six Critical Questions• When should I be concerned?• How can I understand what is happening?• What caused this to happen and can it be

prevented?• Which treatments and interventions will

make will help?• Where can I turn for services?• What does the future hold?

Example• Short-Term Objectives

– Develop, with existing tools, at least one efficient diagnostic instrument (e.g., briefer, less time intensive) that is valid in diverse populations for use in large-scale studies by 2011. (2 years, $2.5 M)

– Validate and improve the sensitivity and specificity of existing screening tools for detecting ASD through studies of the following community populations that are diverse in terms of age, socio-economic status, race, ethnicity and level of functioning by 2012. (3 years, $5.0 M)

• Long-Term Objectives– Validate a panel of biomarkers that separately, or in combination with

behavioral measures, accurately identify, before age 2, one or more subtypes of children at risk for developing ASD by 2014. (5 years, $30 M)

– Develop five measures of behavioral and/or biological heterogeneity in children or adults with ASD, beyond variation in intellectual disability, that clearly relate to etiology and risk, treatment response and/or outcome by 2015. (5 years, $40 M)

– Identify and develop measures to assess at least three continuous dimensions of ASD symptoms and severity that can be used to assess response to intervention for individuals with ASD across the lifespan by 2016. Effectively disseminate at least one valid and efficient diagnostic instrument (e.g., briefer, less time intensive) in general clinical practice by 2016. (5 years, $10 M)

– Effectively disseminate at least one valid and efficient diagnostic instrument (e.g., briefer, less time intensive) in general clinical practice by 2016. (5 years, $5 M)

Thank You!