Project Detail
Interagency Autism Coordinating Committee (IACC) logo
Office of Autism Research Coordination (OARC) logo

Folate rechallenge: A pilot study  

New evidence suggests that autism spectrum disorder (ASD) may be associated with abnormalities in folate metabolism, which is a process that affects genetic expression by facilitating the formation of methyl donors for DNA methylation. Limited data show that some children with ASD show behavioral improvements with folic acid (FA) therapy, while others show a worsening effect. If behavioral worsening is linked with abnormalities in folate metabolism, then nutritional modifications could normalize these processes and result in clinical improvements. To address this premise, a randomized, placebo-controlled crossover pilot study will be conducted. The first phase of the study will focus on the behavioral and biochemical responses of children with ASD to high-dose folic acid supplementation. Because FA requires biochemical conversion to become active, and select genotypes impede this conversion, behavioral improvements may be observed in some children while increased problem behaviors may occur in others. During the second phase, children who had a worsened behavioral response to FA during phase 1 will participate in an open-label trial of high-dose Metafolin® supplementation. Because Metafolin® is an active folate metabolite that should not be affected by genotypes in the folate pathway, this supplement may yield behavioral improvements without the risk for behavioral worsening. Results from this project may provide support for continued study of the potential relationship between folate metabolism and problem behaviors among children with ASD. Project Status


Funder National Institutes of Health
Fiscal Year Funding $10,961.00
Project Number 5M01RR000188-45
Principal Investigator Beaudet, Arthur
Received ARRA Funding? No
Strategic Plan Question Question 4: Which Treatments And Interventions Will Help? (Interventions)
Strategic Plan Objective Yellow dot: Objective has some degree of funding, but less than the recommended amount. 4SC. Test safety and efficacy of at least five widely used interventions (e.g., nutrition, medications, assisted technologies, sensory integration, medical procedures) that have not been rigorously studied for use in ASD by 2012. IACC Recommended Budget: $27,800,000 over 5 years.
Federal or Private? Federal
Institution Baylor College of Medicine
State/Country Texas
Web Link 1 Folate rechallenge: A pilot study (External web link)
Web Link 2 No URL available.
Web Link 3 No URL available.
History/Related Projects Folate rechallenge: A pilot study | $4,578.00 | 2010 | 5M01RR000188-46