|Project Title||Principal Investigator||Institution|
|Clinical Trials Network||Staff Member||Autism Speaks (AS)|
|Randomized phase 2 trial of RAD001 (an MTOR inhibitor) in patients with tuberous sclerosis complex||Sahin, Mustafa||Childrens Hospital Boston|
|The effects of oxytocin on complex social cognition in autism spectrum disorders||Bartz, Jennifer||Mount Sinai School of Medicine|
|Trial of a glutamate antagonist in the treatment of OCD and autistic disorders||Swedo, Susan||National Institutes of Health|
|Association of cholinergic system dysfunction with autistic behavior in fragile X syndrome: Pharmacologic and imaging probes||Reiss, Allan||Stanford University|
|Neuronal nicotinic receptor modulation in the treatment of autism: A pilot trial of mecamylamine||Arnold, L. Eugene||The Ohio State University|
|ACE Center: Understanding repetitive behavior in autism||McCracken, James||University of California, Los Angeles|
|ACE Center: The pharmacogenetics of treatment for insistence sameness in autism||Owley, Thomas||University of Illinois at Chicago|
|Developing treatment, treatment validation, and treatment scope in the setting of an autism clinical trial||Stein, Peter||University of Medicine & Dentistry of New Jersey|
|Developing treatment, treatment validation, and treatment scope in the setting of an autism clinical trial||Johnson, William||University of Medicine & Dentistry of New Jersey - Robert Wood Johnson Medical School|
|Developing treatment, treatment validation, and treatment scope in the setting of an autism clinical trial||Novotny, Sherie||University of Medicine & Dentistry of New Jersey - Robert Wood Johnson Medical School|
|IACC Strategic Plan Objective||2008||2009||2010||2011||2012||Total|
|Complete at least three randomized controlled trials on medications targeting core symptoms in people with ASD of all ages by 2014.
IACC Recommended Budget: $22,200,000 over 5 years
|4.L.A. Funding: The recommended budget was partially met.
Progress: 10-14 studies have been funded, which is more than the minimum recommended, and momentum within the pre-clinical phases of this objective is currently building. It should be noted, however, in that many of these studies are small trials or pilot studies.Â
Remaining Gaps, Needs, and Opportunities: Though there is little evidence that CNS drug development in animals will translate to humans, either in terms of toxicity or efficacy, there is still a need for investment in well-established animal model studies to identify promising molecular, cellular, or systems targets before mounting randomized clinical trials in humans. However, existing drugs for other indications may be adapted to ASD without extensive pre-clinical work, and there is also evidence for proof of concept studies for ASD (particularly those addressing core symptoms). It is also critically important to develop appropriate outcome measures for use in trials.