On December 14, Autism Speaks, together with the American College of Neuropsychopharmacology (ACNP), held a symposium in Boca Raton, Fla., to review the "Development of Novel Neuropharmacological Therapeutics for Autism." The symposium, a satellite meeting of the ACNP's 46th annual meeting, examined the opportunities and barriers to progress in this field. The ACNP is one of the most prestigious organizations dedicated to furthering research and education in neuropsychopharmacology and related fields.
More than 80 scientists from a variety of backgrounds, including clinicians, basic neuroscientists, and researchers from the pharmaceutical industry, participated in the event; in addition to investors and entrepreneurs -– all key figures in hastening drug development for autism.
The symposium was divided into three sessions: Treatment Models, Measures for Diagnostic Assessment and Outcomes and Drug Discovery and Development.
During the first session, Eric Hollander, M.D., from the Mount Sinai School of Medicine reviewed the current status of different classes of pharmacological agents that have been tested in clinical trials for autism; Yale's Lawrence Scahill, Ph.D., discussed the importance of using robust clinical trial design; and Gahan Pandina, Ph.D., of Johnson & Johnson offered an insider view of the process J&J went through to obtain FDA approval for the use of Risperdal in treating ASD. These presentations were complemented by the presentation from Michael Aman from Ohio State University, who talked about rating scales used to diagnose autism and ‘outcome measures' that are informative in detecting meaningful therapeutic benefit of a drug being tested in a clinical trial.
The second session featured discussions of the use of animal models as a vital part of the drug discovery process. Jacqueline Crawley, Ph.D., of the NIMH presented a review focused on the validation of mouse behaviors (e.g. social interactions) relevant to the core symptoms of autism, and how measurement of changes in these mouse behaviors could be used as surrogate markers for preclinical evaluation of new therapeutics. A presentation by Timothy Roberts, Ph.D., from the Children's Hospital of Philadelphia, who showed the application of magnetoencephalography (MEG, a brain imaging technique) to detect altered auditory processing and language impairments in individuals with autism. Dr. Roberts explained that auditory processes are also present in rodents, which could be translated into a new animal model.
The third session focused on developments in more basic neuroscience that point towards novel therapeutic approaches and to the identification and validation of new therapeutic targets. Mark Bear, Ph.D., of MIT reviewed the role of the FMRP protein in Fragile X as a risk factor for autism -- and how pharmacological manipulation of the mGluR5 receptor (a pharmacological target) can overcome the molecular deficits that cause Fragile X. Jeremy Veenstra-VanderWeele, Ph.D., of Vanderbilt University reviewed the findings from genetic research on the causes of autism and highlighted the genetic targets that have been identified. A presentation by Uwe Rudolf, M.D., of Harvard University focused on preclinical studies showing the GABA receptor system (a major inhibitory pathway in the brain) warrants further investigation. Robert Ring, Ph.D., from Wyeth concluded the symposium by reviewing the evidence (preclinical and clinical) supporting manipulation of the oxytocin receptor system as a novel treatment approach. He highlighted some of the difficulties that must be overcome in producing useful medications that interact with neuropeptide system located in the brain.
Autism is a complex disorder that presents with a myriad of psychiatric and behavioral symptoms that can vary in severity. In 2006 the Food and Drug Administration (FDA) approved Risperdal as a treatment for some symptoms of ASD. This was the first, and remains the only, FDA-approved medication indicated to treat ASD. Other medications, such as SSRIs and atypical antipsychotics, are widely used off-label to control other symptoms. No pharmacological interventions are available to directly alleviate the core deficits of autism.
Autism Speaks' Treatment Committee is dedicated to ensuring that robust research is performed to develop new medicines for autism. The Committee will utilze the information presented in this symposium in its efforts to help advance this research.