Minutes of the Interagency Autism Coordinating Committee (IACC) Meeting on May 4, 2009
The Interagency Autism Coordinating Committee (IACC, also referred to as "the committee") convened at 9:06 a.m. on May 4, 2009, in the William H. Natcher Conference Center at the National Institutes of Health in Bethesda, MD, and adjourned at 4:00 p.m.
In accordance with Public Law 92-463, the meeting was open to the public. Thomas R. Insel, M.D., Director, National Institute of Mental Health, chaired the meeting.
Committee Members Present at the Meeting: Thomas R. Insel, M.D., IACC Chair, National Institute of Mental Health (NIMH); Della Hann, Ph.D., Executive Secretary, Office of Autism Research Coordination (OARC), NIMH; Duane F. Alexander, M.D., Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); Ellen W. Blackwell, M.S.W., Centers for Medicare & Medicaid Services (CMS); Judith Cooper, Ph.D. (Representing James F. Battey, M.D, Ph.D.), National Institute on Deafness and Other Communication Disorders (NIDCD); Chris DeGraw, M.D., M.P.H. (Representing Peter van Dyck, M.D., M.P.H.), Health Resources and Services Administration (HRSA); Lee Grossman, Autism Society of America; Gail R. Houle, Ph.D., U.S. Department of Education (ED); Deborah Hirtz, M.D., (Representing Story Landis, Ph.D.); National Institute of Neurological Disorders and Stroke (NINDS); Yvette M. Janvier, M.D., Children's Specialized Hospital; Jennifer G. Johnson, Ed.D., Administration for Children and Families; Cindy Lawler, Ph.D. (Representing Linda Birnbaum, Ph.D., National Institute of Environmental Health Sciences; Christine M. McKee, J.D.; Lyn Redwood, R.N., M.S.N., Coalition for SafeMinds; Cathy Rice, Ph.D., (Representing Edwin Trevathan, M.D., M.P.H.), Centers for Disease Control and Prevention; Stephen M. Shore, Ed.D., Autism Spectrum Consulting; and Alison Tepper-Singer, M.B.A, Autism Science Foundation.
Call to Order and Opening Remarks
Chairman Thomas Insel welcomed the committee and noted that Ms. Kathleen Sebelius had been approved on April 28, 2009, as the new Secretary of Health and Human Services. He said that he hoped she could attend a future meeting of the IACC. Dr. Insel described the flurry of activity around the American Recovery and Reinvestment Act (ARRA), which provided around $10 billion in funding to the National Institutes of Health (NIH). Several Requests for Applications (RFAs) on autism had been released, including a large RFA to study the heterogeneity of autism spectrum disorders (ASD), which reflects priorities outlined in the Strategic Plan for ASD Research. Dr. Insel noted the IACC Strategic Plan for ASD Research was fortuitously released at the same time a large influx of funds became available. He also mentioned that three important papers about autism had recently been published in Nature - one article about how people with autism process biological motion1 and two breakthrough papers on genetic risk factors.2,3 After Dr. Insel's opening remarks, the members of the IACC introduced themselves and Dr. Della Hann informed the committee that the next meeting of the full IACC had been changed to July 15, 2009. During this meeting, the IACC will meet jointly with the National Vaccine Advisory Committee (NVAC) in the morning portion. Dr. Stephen Shore indicated that he had a scheduling conflict and would not be able to attend the July 15th meeting.
Update from the Services Subcommittee
The committee then heard updates from the Services Subcommittee that had last met on February 24, 2009. Ms. Ellen Blackwell, co-chair of the subcommittee, reviewed the Request for Information (RFI) that had been released in the fall of 2008. In total, the RFI soliciting input about service needs for people with ASD received 137 responses. Twenty-one respondents self-identified as having ASD. Ms. Blackwell and Mr. Lee Grossman analyzed the content of the RFI responses and identified the top six categories of feedback. The largest proportion of responses related to concerns about adults with ASD. The second most popular category was community acceptance and inclusion, followed by family support, school services (including the need for more teacher training and education), and provider education and training. The next category was infrastructure, which Ms. Blackwell explained included concerns about systems of care, private insurance, and coordinating care.
Ms. Blackwell said that the Services Subcommittee had discussed using the RFI responses to help generate recommendations about services and supports to send to the Secretary. She said that additional expert work groups might need to be convened to create such a document, ideally to be updated annually. The Services Subcommittee had also decided to hold regular presentations at each of its meetings. CDC and HRSA are slated to make the first presentation on their Learn the Signs. Act Early. program. Dr. Gail Houle will present on the Department of Education's activities at a future meeting. The Subcommittee also discussed collecting a list of federal agencies providing ASD services and supports, as well as identifying lead staff at the agencies.
Mr. Grossman discussed the possibility of conducting an IACC town hall meeting on July 24, 2009, during the annual Autism Society of America conference. He said that he expected a fairly robust turnout of 1,700 to 2,000 participants at the conference. Mr. Grossman said that the town hall meeting might be a good forum to discuss updating the Strategic Plan for ASD Research. He also asked for input on whether such a meeting should be restricted to service-related issues or be open to all issues of the IACC.
Dr. Duane Alexander expressed enthusiasm for the idea of a town hall meeting and said that he felt a focus on services would be most beneficial. Ms. Alison Singer also supported the town hall meeting and suggested that it be used as a platform to inform the services portion of the Strategic Plan for ASD Research. She also suggested that the meeting be made available to those who couldn't attend in person, through webinar or phone conference. Ms. Lyn Redwood voiced her support for a services focus. Mr. Grossman said that while they could try to encourage comments on services, a town hall forum is ultimately open to any topic. The committee unanimously voted in favor of conducting a town hall meeting at the ASA conference.
Before closing, Ms. Blackwell recommended that the IACC hear a presentation on Applied Behavioral Analysis (ABA) at a future meeting. Dr. Insel said that the committee might benefit from hearing about best experiments in policy from other states. Mr. Grossman said that he and Ellen were interested in presentations exploring the broadening definition of ABA, its purpose, and the population that receives the most benefit from specific behavioral interventions.
Update from the Subcommittee fro Planning the Annual Strategic Plan Updating Process
Dr. Insel reported that the Subcommittee for Planning the Annual Strategic Plan Updating Process, (also referred to as the "Planning Subcommittee") had convened for its first meeting on March 17, 2009, to formulate a process for updating the Strategic Plan for ASD Research. The members of the Planning Subcommittee recommended conducting a portfolio analysis in order to identify gaps in funding that need to be addressed. The IACC authorized the Office of Autism Research Coordination (OARC) by e-vote to initiate the portfolio analysis before the May 4 meeting. The analysis is similar to the one that was conducted prior to the initial version of the strategic plan. The Subcommittee also discussed other methods for gathering additional input such as holding expert workshops, town hall meetings, and/or issuing a Request for Information (RFI). Dr. Insel then summarized potential timelines for updating the strategic plan by January 2010.
Ms. Singer recommended that the updating process include consideration of items that had been tabled during the drafting of the initial strategic plan. She said that she felt it was critical to convene scientific workshops before releasing the 2010 update of the strategic plan. Dr. Alexander said that a town hall meeting would not be well-suited for collecting information on updating the plan, but that an RFI could reach a national audience. He suggested using the RFI to highlight the items that had been tabled for the next version of the plan, in addition to potentially convening one scientific workshop. Dr. Janvier recommended asking the chairs of the previous scientific workshops to help identify any recent scientific breakthroughs. She said that she did not think the committee would see significant progress on the goals just set in the strategic plan and that a town hall meeting would not be helpful. Ms. Blackwell commented that the previous scientific workshops were very helpful and Dr. Lawler said that she liked the idea of conducting scientific workshops informed by an RFI. She suggested releasing an RFI before the workshop and compiling the comments for the attendants. Ms. Redwood said that she felt that the scientific workshops were helpful but that four specific workshops were unnecessary. She recommended conducting a meeting by bringing back the previous workshop chairs, leaders in the autism research community, and public stakeholders.
Dr. Insel asked the committee which scientific area they would focus on if only one meeting were held. The previous scientific workshops were held as four one-day sessions on diagnosis, biology, treatment and interventions, and risk factors. Ms. Redwood said that she would still like to see all areas touched upon but in a briefer format. Dr. Insel summarized that the committee did not want a town hall meeting to update the strategic plan but there was enthusiasm for scientific workshop(s) to be held in September and potentially issuing an RFI. He also recommended using a venue like the annual International Meeting for Autism Research (IMFAR) to solicit input from the scientific community in the future. The IACC voted to issue an RFI on updating the strategic plan and convene a scientific workshop. The IACC also authorized the Planning Subcommittee to proceed with revisions to the plan. (Prior to this, the subcommittee was only charged with developing the process with which to update the strategic plan.)
Summary of Advances
Dr. Hann reviewed the progress on the 2008 Summary of Advances, which is to be updated annually according to the Combating Autism Act. The OARC compiled a list of 257 articles based on a broad NIH Library search of peer-reviewed articles and reviews published in 2008 that included the terms "autism" or "autistic." The list was sorted into six scientific or research-related categories that roughly matched chapters in the strategic plan and the IACC members were asked to review the articles and suggest any editions, deletions, or re-categorizations. A revised list was then sent to the committee to select articles from each category for inclusion in the Summary of Advances. Six IACC members (three federal and three public) e-mailed their selections to OARC. One member recommended including all 257 articles in the Summary of Advances. Dr. Hann then presented the list of 120 articles that represented the selection of two or more members and a third option containing 41 articles selected by three or more members of the committee. The counts represent discrete articles; articles could be included in more than one category making the total count higher for each option. Dr. Hann then presented the break-down of articles by category for each option. She also asked for the committee's input about including articles published in 2008 that had been included in the 2007 write-up based on their e-publication date.
Ms. Redwood asked for clarification of the purpose of the Summary of Advances and Dr. Hann said that it was mandated by the Combating Autism Act and was intended for the HHS Secretary and the public. Last year's Summary of Advances was forwarded to the Secretary and made available to the public on the IACC Web site. She confirmed that information on the source of funding, when available, would be part of the final document.
The committee then discussed the merits of the various options. Dr. Alexander supported including 120 articles because the set of 41 articles was skewed, with nearly half of the articles relating to the "risk factors" category. He also supported including the duplicate articles from 2007 so that people would not question why they were not included. Dr. Insel recommended adopting an objective methodology that could be consistently reproduced every year; (for example, only accepting articles from journals with an impact factor over 3). He hoped that the Summary of Advances could be used to measure growth in fulfilling the objectives of the strategic plan.
The committee unanimously voted to include only articles that were formally published in the year of the Summary of Advances (not just an e-publication). This meant that six articles included in the 2007 Summary of Advances would be included in the 2008 document and any publications that were only e-published in 2008 would be stripped from the list. The committee discussed stratifying the articles by journal impact factor, which Dr. Insel explained, is the grade that scientific publications receive based on citation frequency. Journals like Science, Nature, and Cell have very high impact factors. He recommended setting a high standard for the scientific advances given credibility by the IACC. Ms. Redwood was concerned about selecting articles based on journal impact factor because of the bias against emerging science published in lesser-known journals.
Dr. Insel noted that studies published in journals with lower impact factor scores might warrant inclusions if the findings were compelling enough. For example, an interesting recent study published in Molecular Psychiatry found significantly higher levels of melatonin and a melatonin synthesizing enzyme in people with ASD than in their peers.4 This journal received a 2008 impact factor of 12.537, (for reference, the New England Journal of Medicine had an impact factor of 50.017), so selecting articles based solely on journal impact factor could have limitations.
Dr. Insel noted that it was a misconception that less prominent journals would be more likely to include novel findings in the field. Emerging findings are frequently published in top journals and less frequently cited journals often publish the replicated results. Dr. Hirtz suggested including an additional section devoted to emerging research that needs follow-up. Ms. Singer endorsed the idea of getting scientific input from the community at IMFAR next year. She also expressed disappointment that only six members of the committee made selections for the Summary of Advances. Dr. Janvier said that she had been overwhelmed by the sheer number of articles.
Ms. Redwood said that the skewed nature of the list of 41 concerned her. Dr. Insel said that the concentration in one subject area could be an interesting gauge of the lack of meaningful research in other areas. The 2008 Summary of Advances could be used as a baseline to see growth in categories related to the strategic plan. Ms. Blackwell asked the committee to consider the option for selecting the articles based on the journal's impact factor. Dr. Lawler suggested ranking the impact factor of journals within categories because she acknowledged that some areas would have smaller cadres of investigators and subsequently fewer citations.
Ms. Singer said that a smaller number of articles might increase the likelihood that people would not be overwhelmed by the bulk of information. Dr. Hann mentioned that the 2007 document included 55 articles, for comparison. Ms. Diane Buckley described the method for developing the 2007 Summary of Advances which relied on NIH program officers to identify the year's top findings. The IACC then approved the findings and developed the top ten advances for the year. Dr. Alexander suggested that the OARC prune the list of 120 based on their scientific expertise and have the IACC approve the list by e-mail. Dr. Insel countered, saying that the responsibility now rested with the committee to decide which advances should be included. If the committee selected the option to include 41 articles, additional articles could be added later if they were deemed extremely important. Alternatively, articles could be stricken if they were not deemed important enough after the first draft.
Ms. McKee said that she was concerned that the "treatments and interventions," "services and supports," and "outcomes" section were extremely thin in the 41-article option compared to the 120-article option. A smaller percentage of funds are going to these categories so she did not want them to be overlooked. Dr. Insel said that the disparity then mirrored the reality of the areas in which advances were being made. Ms. McKee asked that the document then acknowledge the absence of advances and cite that the committee hopes to see growth in these areas.
Dr. Jennifer Johnson asked how the committee was defining "advance." She wanted to make sure that the committee only endorsed quality research in the Summary of Advances. Ms. Redwood suggested that the full list of 257 articles be made available to the public in addition to the final write-up. Ms. Singer said this was a good compromise between the options. Dr. Insel mentioned that new regulations requiring research using NIH funding be submitted to PubMed Central would mean that future articles would be available in full at no cost to the public. The IACC then voted on the different options for generating the Summary of Advances. The majority of the committee (9 members) voted to include the 41 articles in the Summary of Advances and append the full list of 257 articles that were initially considered. Two committee members voted to include the 120-article selection. The committee agreed that the appended list would link to the article abstracts in PubMed.
Speaker - Susan E. Swedo
Dr. Susan E. Swedo, a senior investigator within the NIMH Intramural program, spoke to the committee about several current projects on autism spectrum disorder being conducted by the Developmental Neuroscience Branch. Dr. Swedo explained that the branch was relatively new and primarily focused on autism rather than the whole spectrum of autistic disorders. Their research efforts are focused on finding a cure and scientists hope to model the same dramatic advances seen in the treatment of leukemia. In treating obsessive-compulsive disorder, intramural researchers conducted careful clinical observations and formed hypotheses about causation and potential treatments for specific subgroups. The same general methodology is applied to researching autism. Multidisciplinary research teams are examining genetic and environmental risk factors for autism. They are also investigating whether the age of the child when exposed to potential triggers affects outcome.
The researchers are able to conduct clinical evaluations and then collaborate with labs within NIH. Dr. Swedo said that one of the strongest collaborations was with Dr. Margarita Raygada and Dr. Owen Rennert at the NICHD. Through the collaboration, they are able to study stem cell models derived from skin biopsies of people with autism. The Developmental Neuroscience Branch also collaborates with Autism Speaks to access their two major networks, the Autism Treatment Network and the Clinical Trials Network.
The Branch's screening study serves as the entry point for all other studies. Over 400 patients have been given a comprehensive diagnostic and behavioral evaluation, a process that takes a minimum of eight hours for each individual. Studies can be grouped into three basic types: the subtype or phenomenologic investigation that looks for clues about etiology by examining groups of people with autism; therapeutic trials testing specific treatments; and hypothesis testing experiments, where findings from other studies can be tested.
Dr. Swedo described a functional magnetic resonance imaging (fMRI) study being conducted by Dr. Marta Gozzi to examine the effects of oxytocin, vasopressin and placebo on emotional processing in autistic individuals. Another study, in collaboration with Dr. Jim Shannon, will use magnetic resonance spectroscopy to evaluate patients' response to treatment with the drug riluzole. Researchers hope that a large study of autistic phenotyping should reveal new information about mechanisms of the disorder. The study enrolls children with autism as early as one year old and follows them for at least three years. Within the study, an investigation of regressive autism revealed that the subgroup is a continuum rather than a dichotomy -- most children experience some signs of regression.
Dr. Swedo described several other studies that had been completed. One study showed that children with autism do not show any specific pattern of dysmorphology (abnormal anatomical characteristics). Another found that children with autism had abnormal overnight electroencephalogram (EEG) readings, showing epileptiform discharges - electrical brain activity similar to that seen in patients with epilepsy. Another sleep study indicated that autistic children have trouble falling into and remaining in rapid eye movement (REM) sleep. Based on this finding, Dr. Ashura Buckley designed a trial of two sleep medications, Donepezil and Articept, to treat the sleep abnormalities. Initial results suggest that the therapies may be effective.
Currently, researchers are also studying the effects of the antibiotic minocycline on the inflammatory response in people with autism. Another study will investigate remittent autism, comparing the characteristics of 40 children whose symptoms remitted to 40 children who continue to have symptoms. This study is actively recruiting and Dr. Swedo said that referrals for participants would be greatly appreciated.
Ms. Blackwell asked Dr. Swedo if intramural researchers were working with autistic adolescents or young adults. Dr. Swedo said that the remitted autism study would include older participants and the fMRI study would exclusively include young adults. Ms. Singer asked if the increases in REM sleep for children in the Donepezil study translated into behavioral improvements. Dr. Swedo said they had, but they were hesitant to link the improvements to the medication because of the relatively intensive nature of the study and the short time period.
Ms. Redwood asked for additional details about the remittent autism study and Dr. Swedo said that the study is enrolling children ages 8 to 18 years old. All travel costs for patients and their families would be paid by NIH. More information about participating in this or other studies at the NIH Clinical Center can be found at:Phone: 1-866-444-6676
TTY: 1-866-411-1010 (toll free)
Website: Clinical Studies
Dr. Duane Alexander commended the caliber of the research being conducted by the intramural program and the speed with which it was organized.
Approval of Minutes from Past IACC Full Committee Meetings
Dr. Insel asked the committee for comments and suggested edits to the minutes from the November 2008, December 2008, January 2009, and February 2009 meetings of the IACC. The minutes had been e-mailed to the committee members prior to the meeting for their review. Ms. Redwood asked if the minutes along with the transcript would be available on the IACC Web site and was informed that they would be. Ms. Singer asked that the name of the advertising agency BBD&O be corrected in the January minutes. The minutes were then approved by vote of the committee.
Speaker - Joanne M. Cafiero, Ph.D.
Dr. Insel introduced Dr. Cafiero, whose current research focuses on how autistic children with severe communication impairments learn language. First, Dr. Cafiero presented some of the myths surrounding autism spectrum disorders and augmentative and alternative communication (AAC). She said that the first misconception was that AAC will inhibit the development of speech. The next is that people with speech do not need any form of AAC. Other misconceptions include the belief that people with ASD will automatically use AAC if it is provided and that AAC is not a viable intervention if it is not used within a specific time frame. Finally, she said that it is a myth that no-tech or low-tech interventions are the best way to start.
AAC unlocks language that is not able to be expressed by the individual, as well as promoting language development. Different forms of AAC may work better for different people depending on their cognitive ability, level of existing language, and fine motor function. AAC ranges from unaided forms of communication like sign language, to low-tech forms using picture boards, to sophisticated high-tech computerized devices. Dr. Cafiero described an analysis of aided (using a device) versus unaided AAC which found that aided AAC required fewer fine motor skills and is more readily comprehensible, although less portable. Importantly, other studies have found that AAC does not inhibit speech production.
There are several forms of evidence-based practices in AAC. Functional communication training (FCT) deters aberrant behavior through communication and has been shown to be an effective strategy. For example, if a child displays aggression when they need a break, they are taught to communicate that need through a picture or sign. The Picture Exchange Communication System (PECS) is an expressive language strategy in which the student exchanges a symbol of an item for the desired item. Speech-generating devices can be used alone or with other forms of AAC. The devices range in sophistication but all model speech for the user. In the augmented input model, the communication partner is also an essential user of the device. Researchers agree that for all forms of AAC, the communication partner needs to model the use of the device for the student. One of Dr. Cafiero's studies found that greater parental input (usage of the AAC device) generated more output from the autistic child.
Dr. Cafiero described the case of a five-year-old autistic boy with self-injurious behavior who was given a high-tech AAC device. After receiving the device, the boy's vocabulary grew and his head-banging behavior decreased. Dr. Cafiero then mentioned ProLoquo2Go an inexpensive AAC application that loads onto an iPhone. She emphasized the need to receive feedback about specific forms of AAC from members of the autistic community. Ultimately the goal is to match an individual with the device best suited to support long-term language development.
After the presentation concluded, Dr. Shore commented on the importance of assisted communication.
Speaker - Dora Raymaker
Ms. Dora Raymaker, an autistic self-advocate, then presented to the committee via phone using a speech-generating device. Ms. Raymaker, co-director of the Academic Autistic Spectrum Partnership in Research and Education (AASPIRE), explained the critical nature of communication. Communication is not speech, she explained. For some, speech may impede or even prohibit the ability to communication. Communication allows an individual to express his desires and to be empowered. It is a dynamic interaction between two or more people.
Ms. Raymaker explained that autistic people may have sensory differences that may impede the person's ability to decode auditory information. Others may have problem speaking words because of problems with oral motor skills. Using AAC can change the mode of communication, from speech and hearing to reading and writing. AAC can also change the timing of communication by slowing the rate of a real-time exchange. AAC can also act as a bridge between one type of communication to another, such as typing to speaking.
Ms. Raymaker described the variety of AAC devices and their variable roles. She said that funding was needed to design and match the devices that are most appropriate for different people's varying needs. AAC technology must be evaluated more thoroughly and input from autistic individuals should be taken under consideration. Price continues to be a barrier for many people - the training to use the device, in addition to the device itself, are often costly. Some systems may also limit individuals' access to AAC. For example, some schools will not allow AAC devices to be taken home, effectively limiting communication after school hours. She closed by reminding the audience that her presentation was made possible by augmentative and alternative communication technology. She also asked that Secretary Sebelius choose to include a member of the organized autistic advocacy community on the IACC.
Dr. Cafiero and Ms. Raymaker then took questions from the committee. Ms. Christine McKee asked Dr. Cafiero what current research in the field excited her. Dr. Cafiero said she was fascinated by the ways people with ASD were using AAC devices to say novel things. For instance, a boy wanted to talk about a tree that had been blown down on a golf course and used the phrase 'golf horse' because he had no way to communicate 'course.' Dr. Cafiero went on to say that providing literary instruction for people with ASD is critical because literacy and AAC are irrevocably woven together.
Dr. Insel asked if any age was too young to begin using AAC. Dr. Cafiero said that a researcher at Penn State was introducing devices to 18-month-olds, so there was neither too young nor too old of an age to begin AAC. Dr. Insel then asked if the presence or absence of speech (or any other variable) predicted who would be successful at using AAC. Dr. Cafiero said that the answer to that was unknown but that she personally supported everyone with autism using AAC suited to his or her needs as early as possible. In follow-up, Dr. Rice asked how one chooses the most appropriate communication device for an individual's needs. Dr. Cafiero responded that, for now, choosing the best device is a trial and error process. Cost can also be a consideration - currently, the ProLoquo2Go costs about $400 (including the iPod touch or iPhone), compared to about $8,000 for a comparable device.
Dr. Cafiero said that the varying architecture of different devices continues to be a major technological hurdle. These differences make learning different devices harder for practitioners who must know the device like their own voice. Ms. Raymaker responded via e-mail to a question posed by an IACC member regarding the means she used to communicate that day. She said that she had used the speech software ProLoquo and the 'Heather' voice on her Mac computer during her presentation. Ms. McKee commented that she had found it frustrating that most companies sell software that can only be used on their own device. One company is now selling their software separately so it can be loaded onto laptops around the house. Dr. Insel mentioned that NIH is required to spend around 2.3 percent of their budget on small companies through Small Business Innovation Research (SBIR) grants. Funding could be put into developing the next generation of AAC technology.
Speaker - Josephine P. Briggs, M.D.
The committee then heard a presentation from Dr. Josephine P. Briggs, Director of the National Center for Complementary and Alternative Medicine (NCCAM). She started by praising the IACC Strategic Plan for ASD Research and then described her own background as a kidney doctor. NCCAM is about ten years old and was created to explore complementary and alternative healing practices using rigorous scientific methods. The center also supports the development of a trained scientific community and disseminates authoritative information to the public. She recommended that the committee visit NCCAM's Web site, saying that it was the most widely-accessed site on complementary and alternative medicine on the Internet.
NCCAM currently receives about 0.4 percent of NIH total budget of $30 billion and often partners with other NIH institutes to conduct research. For instance, NCCAM recently partnered with the National Institute of Aging and others to conduct a large study of gingko's effect on Alzheimer's disease. The study failed to demonstrate any benefit of gingko on preventing cognitive decline in a high-risk group. More ambiguous results came from a study of glucosamine and arthritis. The glucosamine had a small amount of benefit for certain subgroups but did not show clear benefits. Another NCCAM study partnered with the National Institute of Mental Health (NIMH) to investigate the use of St. John's Wort to treat major depression.
Dr. Briggs then presented the results of the National Health Interview Survey, last conducted in 2007 by the Centers for Disease Control and Prevention (CDC). The survey found that 40 percent of Americans are using some form of complementary or alternative medicine (CAM). CAM includes non-vitamin, non-mineral dietary supplements like fish oil and Echinacea, as well as relaxation techniques like deep breathing, yoga, and meditation.
About 60 percent of women and 30 percent of men use CAM. The survey showed an increase in the number of children using CAM, with one in nine children using some form. Overall, people with more education were more likely to use CAM.
Dr. Briggs said that the most common reason cited for using CAM was 'wanting to be healthier.' It is mostly used in addition to conventional care, not as a replacement. Most people using CAM cite back pain and other kinds of pain. The survey included 54 children with autism out of the representative sample of 9,000 children, however this subset was too small to analyze separately.
NCCAM studies can impact the sale of supplements. Echinacea sales dropped off precipitously after a study failed to document any clear impact of the frequency and duration of colds. Alternately, sales of fish oil and omega-3 supplements jumped upon evidence of some benefit for cardiovascular health.
She said that NCCAM's small size was a challenge. Additionally, most supplements are compounds without identified biological targets which poses problems during study. For example, Echinacea can differ markedly depending upon how it is extracted. The study showing no immune benefit was criticized for not using the "ideal" preparation. Before undertaking a major randomized clinical trial of a CAM intervention, Dr. Briggs said that researchers would like to have a strong biological hypothesis, a well-described intervention, proof of concept, preliminary clinical data, chemistry of any natural products, and very sensitive outcome measures.
She said that NCCAM has documented the positive benefits of mindfulness meditation for stress reduction and quality of life. NCCAM has also invested heavily in placebo research and areas of pain management. She said that many parents of children with autism used CAM and that the safety of the child must be viewed as paramount. She emphasized that evidence base must be incremental - just because a supplement or technique is currently in use does not mean that a large randomized control trial (RCT) should be launched. Dr. Briggs explained that RCTs will only detect large effects on well-targeted populations. In a complex problem, researchers may be left with no clear answer.
She also addressed how anecdotal evidence can be misleading. She warned that many people, particularly those trying to help their children cope with disorders, may be vulnerable to misplaced hopes and expectations. Therefore, objective approaches are needed. Currently, NCCAM is funding a small pilot study examining the effects of Omega-3 fatty acids compared to placebo in children with ASD.
Dr. Briggs spoke about 'N of one' trials to guide therapeutic decisions by physicians. For example, someone might work with a physician to compare glucosamine with a placebo to treat his knee pain. By alternately using the placebo or the glucosamine for two-month blocks, with three intervals of each, he or she should be able to reliably determine the personal effectiveness of the intervention. This assumes that the intervention will show an effect in a short interval and will leave the system quickly when not being used. One barrier for the 'N of one' trial is the ability to make placebos indiscernible from the treatment.
Ms. Redwood cited statistics showing a very high use of CAM for individuals with ASD and emphasized the importance of defining subgroups that might respond to specific therapies. She said that studies of methylcobalamin should be given to the subgroup of autistic children with low or high levels of methylmalonic acid in their urine, rather than testing the supplement on any child with autism. Ms. Redwood asked if the omega-3 pilot study specifically targeted children with low levels. Dr. Briggs said that while omega-3 red cell membrane composition had been tested, the pilot study did not target a specific subgroup.
Dr. Insel asked when a treatment changed from being complementary to mainstream. Dr. Briggs responded that there was not an established answer to that question but that it was largely based on whether the practices originated from outside the mainstream.
The committee then heard public comment from four speakers. Ms. Theresa Wrangham, President of SafeMinds, spoke first. She asked that the committee expand the number of treatment studies in the Strategic Plan for ASD Research, as well as the amount of environmental research. She recommended the expansion of the number of projects studying metabolic and/or immune interaction mechanisms with the central nervous system. She also recommended conducting active screening prevalence studies of people born before and after 1987. She requested that the IACC convene an independent panel to address vaccine safety concerns.
Mr. Ari Ne'eman then testified on behalf of the Autistic Self-Advocacy Network (ASAN). He asked that a member of the organized self-advocacy community be represented on the committee and called for more funding for quality of life and services research in the strategic plan. He urged the committee to move beyond the focus on autism causation and cure. This should include research on health care disparities for autistic youth and adults. He also asked that the committee address bioethical issues associated with prenatal testing for ASD.
Ms. Katie Miller then spoke before the committee and applauded the presentations on AAC, saying it was a critical area that was vastly underfunded. She echoed the request that the IACC add a member from the organized self-advocacy community and that the strategic plan reflect more research on quality of life and services. She requested that the disorder not be compared to cancer and asked that people stop using language describing the disorder as an "epidemic" or people with ASD as a "burden." Finally, she asked that the committee consider the ethical implications of prenatal testing for ASD.
Ms. Paula Durbin-Westby asked that more funding be allocated to AAC research and asked that this money be diverted from the funding for genetic and treatment research, not from the minimal funding for service-related work. She recommended that autistic adults be included as collaborators and co-researchers in studies of autism. She also requested that additional autistic self-advocates be added to the committee.
Closing Comments and Adjournment
Dr. Insel thanked those who had made public comment and reminded the committee that only the HHS Secretary retains the authority to make changes to IACC membership. He said he hoped Ms. Sebelius would be able to attend a meeting in the near future. The meeting was then adjourned.
These minutes of the IACC full Committee Meeting on May 4, 2009 were approved by vote of the IACC on July 15, 2009.
I hereby certify that this meeting summary is accurate and complete.
Thomas Insel, M.D. /s/
Chair, Interagency Autism Coordinating Committee