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Statement of Owen Rennert, M.D.
Scientific Director
National Institute of Child Health and Human Development
National Institutes of Health
Committee on Government Reform
U.S. House of Representatives
Washington, D.C.
April 26, 2001

Autism – Why the Increased Rates? A One Year Update

Mr. Chairman and Members of the Committee, I am Dr. Owen Rennert, Scientific Director of the National Institute of Child Health and Human Development (NICHD) at the National Institutes of Health (NIH). I appreciate this opportunity to provide information, on the behalf of the NIH Autism Coordinating Committee (NIH/ACC), about ongoing and planned research activities at the NIH that are relevant to autism and pervasive developmental disorders. The NIH/ACC comprises the National Institute of Mental Health (NIMH), NICHD, the National Institute of Neurological Disorders and Stroke (NINDS), the National Institute on Deafness and Other Communication Disorders (NIDCD), and the National Institute of Environmental Health Sciences (NIEHS).

Autism is a cruel disorder, not only as a result of the disability it causes, but also because it is an illness that challenges the emotional bond between child and parent. The family must watch an apparently healthy child slip away, and succumb to a brain disorder that, in its most severe forms, effectively isolates that child -- socially, cognitively, emotionally, and linguistically – denying other family members even the opportunity to console and comfort. It is a disorder that completely changes the rhythms of family life, putting enormous strain not only on parents, but on siblings. And of course, autism does not end in childhood. Often symptoms become even more difficult to manage as the person with autism ages. In light of these immense human costs and the significant public health burden that autism brings with it, the Institutes that comprise the NIH/ACC are working to focus the research community with ever-greater intensity on this terrible disease, and we appreciate continued parent involvement in our efforts.

Last October, Congress passed The Children's Health Act of 2000 (P.L. 106-310). Section 101 of the law called for expansion, intensification, and coordination of autism-related scientific programs at NIH. I am pleased to report the significant progress that we are making, including progress toward the establishment of a new network of Centers of Excellence in Autism Research. This new network of major research centers will bring disciplines including developmental neurobiology, genetics, and psychopharmacology to bear on questions about the causes, early detection and diagnosis of autism, as well as treatment and prevention. The new law calls for greater efforts to collect and share genetic materials and tissue samples, and to provide a means through which the public may both obtain and provide information to the Director of NIH. I will describe our progress toward these ends.

Congress accurately recognized the broad impact of these disorders and directed the Secretary of Health and Human Services to establish an Interagency Autism Coordinating Committee (IACC). The IACC will include NIH, the Centers for Disease Control and Prevention (CDC), and other HHS agencies. Most recently, the Agency for Healthcare Research and Quality and the Agency for Toxic Substances and Disease Registry have requested membership. The law also calls for other Federal departments – for example, the Department of Education – to be represented on the Committee. Dr. Ruth Kirschstein, the Acting Director of NIH, has asked Secretary Thompson to delegate to NIH authority for establishing the IACC. The Act also authorizes the Secretary, at his discretion, to appoint parents or other legal guardians of individuals with autism or other pervasive developmental disorders to the IACC.

Autism Research Initiatives at the NIH

In the few years since the NIH/ACC was established, there has been considerable expansion and enhanced coordination of autism research efforts at NIH. The amount of NIH support of autism related research grew from $22 million in Fiscal Year 1997 to $40 million in FY 1999, nearly doubling in a period of two years. In FY 2000, funding again increased substantially, to approximately $52 million, a sum that encompasses a large number of grants, contracts, and intramural research programs distributed across the NIH. Indeed, this continuing growth demonstrates the commitment of the Institute members of the NIH/ACC to the broad intensification of autism research efforts called for in the Children’s Health Act. Most of these ongoing NIH-supported projects are investigator-initiated. Nonetheless, in many cases, NIH actively has taken steps to encourage, structure, and support autism research programs that were ultimately funded. As you requested, Mr. Chairman, I am supplying for the record the 10-year funding history of NIH-sponsored autism related research, and the list of projects funded in FY 2000.

Autism Treatment RFA. Recently, the NIH/ACC has undertaken a new research initiative in the realm of treatment interventions directed at autism symptoms. In November 2000, as a follow-up to a major meeting that they had hosted and supported, the NIH/ACC Institutes issued a Request for Applications (RFA) to solicit research applications on innovative approaches to the treatment of autism. The Institutes have set aside $1,000,000 to fund innovative autism treatment proposals that peer reviewers regard as the most promising. Approximately 30 applications have been received and are scheduled for peer review at the September National Advisory Council meetings.

Reissuance of Autism Program Announcement. In February 2001, the five member Institutes of the NIH/ACC reissued a Program Announcement entitled "Research on Autism and Autism Spectrum Disorders." This announcement clearly states for the research community NIH's continuing interest in and support of research dealing with these disorders.

Centers of Excellence in Autism Research. There are currently no NIH-funded Centers with a primary focus on autism or autism spectrum disorders. The Center grant mechanism, which is currently available to teams of researchers who wish to apply, provides support for multidisciplinary and multi-investigator studies of a specific research problem of a complex nature that requires the application of diverse expertise and methodologies.

As noted above, a major component of the autism provisions of the Children’s Health Act of 2000 is the Centers of Excellence in Autism Research program. The NIH/ACC is implementing a strategy for developing these centers. As part of these efforts, the NIH/ACC Institutes intend to release an RFA containing set aside funds for research support for the development of center applications. This is the first stage of an overall plan to support a variety of investigative teams – and whenever possible, to recruit the participation of outstanding investigators who previously have not worked in autism research – in order to maximize the probability that they will become highly qualified applicants for the Centers of Excellence in Autism Research. Each developmental award under this initial RFA will be for one year and a maximum of $100,000 for direct costs. The estimated total funds (direct and indirect costs) available for support for all awards made under the center development grants RFA will be $1.5 million, permitting as many as ten awards, the first of which could be funded in September 2001, if meritorious grant applications are submitted. Let me emphasize that participation in this RFA will not itself be a factor in the review of Center applications. Current grantees, for example, may decide to submit a Center application without participating in this developmental RFA.

A second RFA will be issued in Fiscal Year 2002 to solicit applications for the Centers of Excellence, with funding of the first of these centers early in FY 2003. NIH anticipates that a pool of approximately $8 million per year will be available for the first 5 years, with subsequent investments depending on the sustained quality of applications. Participating NIH Institutes will collaborate in funding the Centers so that they will become an interactive network of high-quality research enterprises covering a spectrum of interests in this disorder.

Brain tissue and genetics resources. The Children’s Health Act of 2000 calls upon NIMH to take the lead in providing for a program under which samples of tissues and genetic materials are donated, collected, preserved, and made available for autism research. Post-mortem brain tissue, offers a unique, high-resolution window into the inner workings of brain cells. For example, by using radioactive tracers on sections of brain tissue, scientists can detect and pinpoint any abnormal activity. Only with access to brain tissue can the underlying neuropathology of autism be uncovered. To take advantage of emerging opportunities for discovery in post-mortem tissue made possible by the new molecular methodologies, NIH, in collaboration with the autism advocacy community, has stepped up efforts to expand brain bank collections for the study of autism.

NIH supports ongoing efforts by the Harvard Brain Tissue Resource Center, UCLA’s West Los Angeles VA Medical Center, and the University of Miami’s tissue bank to collect and make this vital resource available to researchers. Recently, special supplements were awarded to target acquisition of necessary biologic materials from individuals with autism for focused study. The work of the brain banks is coordinated by the Autism Tissue Program through the National Alliance for Autism Research and, of course, through the commitment of families who arrange for tissue donations when individuals with autism die.

Some of the most important clues to the biology of autism will come from genetics. Twin and family studies have shown that genes play a substantial role in autism risk. These studies show that genetic relatedness to a person with autism increases the risk of autism substantially -- indeed far more than is observed in other serious brain diseases such as schizophrenia or manic depressive illness -- and much more than many general medical illnesses, such as type 2 diabetes, that are widely understood to have a component of risk due to genetic susceptibility. However, there is substantial evidence that risk for developing autism does not appear to be due to a single gene in most families, but instead to the interaction of multiple genes. At the clinical level, this explains why autism has a complex pattern of inheritance. For scientists, however, it means that any individual gene may contribute only a small increment of risk, and is thus very difficult to isolate. One of the key needs to solve the genetics of disorders such as autism is a large enough collection of samples. Supported under a contract with Washington University, the NIMH Center for Genetic Studies at Rutgers University has been receiving data and blood samples from NIMH-funded autism genetics projects at Stanford University, New England Medical Center, Vanderbilt University, and the University of North Carolina. These data and biomaterials are widely distributed to the scientific community to conduct analyses on the genetic basis of autism, and their availability is expected to accelerate collaborations among researchers and the discovery of genes producing disease vulnerability.

Network on the Neurobiology and Genetics of Autism: Collaborative Programs of Excellence in Autism (CPEAs). In 1997, in response to the recommendations of the Autism: State of the Science Conference held in 1995, and through an RFA, the NICHD, with co-funding from the NIDCD, established the Network on the Neurobiology and Genetics of Autism, referred to as the CPEAs. Each multidisciplinary, often multi-site project is studying some particular basic and clinical aspect of the biological etiology (including possible genetic, immunological, and/or environmental causes), brain structure and function, and clinical course of autism. Each multidisciplinary project has a unique focus and research plan. In addition, all projects use a common diagnostic protocol and common core measures and procedures to collectively address some research questions that are beyond the resources and/or subjects of any single project. Individually or collectively, the CPEAs investigate the causes, diagnosis, early detection, prevention, and treatment of autism. Expertise at the CPEAs ranges from immunology, molecular genetics, and developmental biology, to clinical and developmental pharmacology. The Network also participates in an international autism genetics research consortium that pursues autism research of international import. An RFA to recompete the CPEAs Network will be issued in October 2001, with five years of funding to be awarded.

Coordination of information across CPEAs occurs through regular meetings of the Network Steering Committee. These annual scientific meetings include investigators from all projects and ongoing subcommittee working groups that discuss topics such as genetics, cognitive development, and communication, supplemented with e-mail and telephone conferencing among the directors of the CPEAs. Extensive outreach and sharing of strategies for recruitment and retention make individuals aware of opportunities to participate in the CPEA research projects. Currently, we have enrolled nearly 2,300 people with well-diagnosed cases of autism in the network and are gathering genetic data from their families. Both competitive and administrative funding supplements to these projects have allowed NICHD to take advantage of this shared resource in facilitating the development of methods in genetic analysis, neuroimaging, neuropsychology, and the conduct of clinical research studies.

A major ongoing CPEA initiative that is co-funded by NICHD, NIDCD, and CDC is the Autism Regression/Vaccination Study. A principal goal of the study is to assess temporal association between measles/mumps/rubella (MMR) vaccine and onset of autism, differentiating early- and late-onset forms of the disorder. Another aim of the study is to try to replicate studies of persistent measles infection in autism cases versus healthy controls, which have been widely publicized but remain unproven. Stage 1 of the project, which got underway in September 2000, includes 1,600 well-diagnosed cases of autism and 1,250 healthy controls; individual vaccination records as well as records of onset of autism, specifically looking at age of onset, age of recognition, and age of diagnosis will be examined. Stage 2 of this project will attempt to replicate previously reported findings regarding abnormal measles antibody titers and persistent measles infections. In this phase, investigators will examine 250 early onset autism cases, 250 regressive autism cases (that is, children who develop normally early on, but subsequently regress), 250 healthy controls matched to early onset cases, and 250 healthy controls matched to regressive autism cases.

Treatment Research

Neuroscience research to understand the pathogenesis of autism is the most promising approach to ultimately developing targeted effective treatments. Until the brain mechanisms responsible for the manifestations of autism are understood, it will not be possible to develop truly targeted interventions that can be expected to correct the core features of the disorder.

While neuroscience research on autism continues, treatment research is currently focused on studying the efficacy and safety of promising treatment interventions which are commonly used in the community without adequate testing or are aimed at specific impairing symptoms such as compulsions, stereotyped behavior, overactivity and self-injurious and aggressive behavior. Both psychosocial and pharmacological interventions are being studied.

Medications trials in autism are ongoing in the Research Units on Pediatric Psychopharmacology (RUPP), a research network supported by NIMH contracts and devoted to testing promising pharmacological agents for the treatment of children and adolescents with autism and other pervasive developmental disorders. Two protocols aimed at testing the efficacy and safety of risperidone in the management of youths with autism and suffering from severe agitation, self-injury and other impairing behaviors have recently completed enrollment. The statistical analyses are scheduled to be done this summer, and will be peer reviewed before being released to the public later this year. Another protocol to test the possible therapeutic benefits of medications in the management of hyperactivity and impulsive behavior in children with autism and other pervasive developmental disorders is starting at five RUPP sites. Even if these studies cannot be expected to provide treatment that will ‘cure’ the core features of autism, they will provide useful data that will guide families and clinicians in making treatment choices for individuals with autism who are severely impaired.

Research on Environmental Exposures. Public concern is mounting over the potential relevance of environmental exposures, either before birth or in early postnatal life, to the etiology of autism spectrum disorders. Last October, neuroscientists, including autism researchers, parent advocates, and NIH program staff were invited to participate in a one-day "brainstorming session" on the role of the environment in autism, organized by the NIEHS. The group identified several key research priorities: large-scale epidemiological studies to determine autism incidence and prevalence trends, studies to describe the natural history of autism and to identify meaningful subgroups that may be at increased risk from environmental exposures, and studies to examine the proposed association between regressive autism and thimerosal in vaccines.

The increased interest in the potential linkage between environmental agents and autism coincided with the issuance of a request for applications by the NIEHS to expand their existing network of Centers for Children’s Environmental Health and Disease Prevention. The Centers program is funded jointly by the NIEHS and the Environmental Protection Administration. The goal of the latest expansion effort is to increase the number of Centers that study the relation between environmental agents and developmental disorders.

With respect to the broad question of thimerosal-containing vaccines, the National Institute of Allergies and Infectious Diseases (NIAID) currently is supporting several hypothesis-based studies in humans, primates and rodents related to thimerosal and mercury-related compounds. One project being conducted at the University of Rochester Vaccine Treatment and Evaluation Unit (VTEU) and the National Naval Medical Center is designed to: a) determine the level of mercury in the serum, hair and urine of children receiving currently recommended childhood immunizations; and b) compare levels of mercury in children who received vaccines containing thimerosal with those receiving thimerosal-free vaccines. The study was initiated in February 2000. Sixty-three infants were enrolled. Preliminary data show that children who received vaccines with thimerosal did not have more mercury in their blood than children who received vaccines without thimerosal. The investigators are currently conducting final analysis of the data in preparation for publication.

Two studies in rhesus macaques were initiated in November 2000 at the University of Washington. These two studies are designed to: a) determine the pharmacokinetics of thimerosal and methyl mercury in serum and brain tissue in juvenile macaques; and b) examine the metabolism/excretion of methyl and ethyl mercury in infant macaques, as well as deposits in hair/fur during the suckling period.

In collaboration with the NIEHS, NIAID initiated three rodent studies in November 2000. These three studies are designed to: a) compare the distribution of mercury levels in multiple organs after administration of thimerosal, ethyl mercury, and methyl mercury; b) evaluate the cellular patterns of distribution of different forms of organic mercury in the brain after administration of thimerosal, ethyl mercury and methyl mercury; and c) evaluate the role of immune activation in altering brain levels of mercury after exposure to thimerosal.

To address environmental exposures more broadly, the Children’s Health Act of 2000 authorized NICHD, along with a consortium of federal agencies including other NIH institutes, the CDC and the EPA, to conduct a major longitudinal study to "investigate basic mechanisms of developmental disorders and environmental factors, both risk and protective, that influence health and developmental processes." In this context, "environment" is defined to include chemical, physical and social-behavioral influences on children, who have critical windows of vulnerability during development, during which time environmental exposures could have a greater influence. A prospective longitudinal study of this magnitude is necessary for answering many questions about childhood diseases and disorders that appear to be increasing, such as autism and asthma. Planning for this study, which will follow about 100,000 children across the U.S. from before birth into adulthood, is currently underway, with pilot studies scheduled for FY 2002-03.

NIH/ACC Annual Scientific Meeting. Each year, the five participating NIH/ACC Institutes organize and convene this conference to focus on a specific, timely topic. The NICHD and NIEHS have taken the lead in developing this year’s session on "Potential Cellular and Molecular Mechanisms in Autism and Related Disorders." One theme of particular interest at the meeting, which is scheduled for September 6-7, 2001, will be the development of new animal models and methodologies to study autism and relevant environmental insults to the developing nervous system.

Mechanisms for Public Input. NIH is committed to bringing public views to its activities, programs and decision-making; to conveying information about NIH's processes and progress to a broad public; and to seeking comment about its operations and help evaluating its performance. Each year since 1998 the Directors and scientific program staff of the Institutes comprising the ACC have held a special meeting to invite representatives of autism research advocacy groups to meet with them, and other institute staff, to openly discuss ongoing efforts and future plans with regard to autism. This year, the meeting was held on March 30, 2001. The meeting was well attended, with representatives of all of the major national groups with an interest in NIH activities with respect to autism represented. In addition to the Directors and staff of the NIH/ACC member Institutes, representatives from other NIH Institutes, PHS agencies, and the Department of Education were also present. Frank discussions were conducted regarding many current issues relating to autism research, and over a dozen presentations were made by non-Federal organizations to share their research findings and points of view.

Other current opportunities for public participation include the NIH Director’s Council of Public Representatives (COPR) meetings, the individual institute advisory council meetings, and specially conducted public forums around the country. In addition, when appropriate, public reviewers are included to bring their insights and perspectives to Scientific Review Groups for research grant applications.

Public Liaison Offices were formally established within each Institute and Center and in the Office of the NIH Director in 1999. They conduct outreach to constituency groups and serve as a contact point for the public, especially with regard to policy matters. The Office of Public Liaison is also the central point within an institute where members of Congress can refer their constituents. For the last two years, the Information and Public Liaison Officers have held a special meeting with members of the autism advocacy community to exchange information. As a direct result, a listserv has been developed with e-mail addresses of advocacy group members and others who wish to subscribe. AUTISM&LIST.NIH.GOV is a valuable resource for anyone interested in up-to-date information about autism related research activities at NIH. Announcements on the listserv will be automatically archived and the list of available messages also will be available in digest form. The NIH Medline Web site for autism was another significant topic of discussion and increased effort in response to the parent meeting last year (medlineplus.nlm.nih.gov/medlineplus/autism.html). This is a searchable site with numerous links related to the latest news, research, scientific literature, autism advocacy organizations, rehabilitation, specific conditions such as Asperger's, related issues such as vaccines, treatment, and the specific NIH/ACC Institutes, which also improved their individual Web sites to provide better information on autism and autism spectrum disorders.

Mr. Chairman, we at NIH have a deep commitment to pursuing the research necessary to defeat autism. We firmly believe that this can be done most effectively by enlisting a larger pool of scientists prepared to do the complex work that is essential for success. That is what we are planning to do with our carefully constructed plan to implement the Children’s Health Act of 2000 passed by Congress last year. We at NIH understand the passion of the parents and families of those who have been affected by autism and related disorders, and share your concern for quickly unraveling the mystery of autism. We look forward to working with the scientific community, the Congress, and parents to address this devastating disorder.

I will be pleased to respond to any questions that you and the other members of the Committee may have. Thank you.

Last revised: May 30, 2001