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Interagency Autism Coordinating Committee (IACC)
Autism Research Database
Office of Autism Research Coordination (OARC)
 
Project Element Element Description

Project Title

Project Title

Efficacy of N-acetyl cysteine in autism

Principal Investigator

Principal Investigator

Berk, Michael

Description

Description

There are few pharmacological options to treat autism, and the currently available drugs target only specific symptoms, such as disruptive behavior. Several lines of evidence suggest the presence of high levels of oxidative stress in autism: reduced levels of glutathione, the brain’s predominant antioxidant; oxidative damage to lipids and other cellular components; and differences in antioxidant gene expression between people with autism and controls. In other neuropsychiatric disorders with similar patterns of oxidative stress, clinical trials on the use of the antioxidant N-acetyl cysteine (NAC), which boosts glutathione, have shown promising results. NAC also impacts the neurotransmitter glutamate, and changes in glutamate levels are also seen in autism. NAC has an excellent tolerability profile, and is a relatively natural treatment option that is currently available as a nutritional supplement. A pilot study of NAC treatment for autism has shown both a reduction in irritability and good tolerability of the compound. These promising results need to be replicated in a larger and more definitive study. Michael Berk and his team at Deakin University in Melbourne, Australia are conducting a randomized, placebo-controlled clinical trial of NAC with 80 children who have recently received a diagnosis of autism. They plan to administer a daily oral dose of NAC for six months, in order to test the efficacy and tolerability of NAC in treating core symptoms of the disorder. If NAC proves to be superior to the placebo in improving autism symptoms, this could have a significant effect on the treatment of autism. This research project may also provide new insights into the pathophysiology of autism and targets for future drug development.

Funder

Funder

Simons Foundation

Fiscal Year Funding

Fiscal Year Funding

0

Current Award Period

Current Award Period

2012-2014

Strategic Plan Question

Strategic Plan Question

Question 4: Which Treatments And Interventions Will Help?

Strategic Plan Objective

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.

Project Link

Project Link

Efficacy of N-acetyl cysteine in autism (External web link)

Institution

Institution

Deakin University

State/Country

State/Country

Australia

Project Number

Project Number

201473

Federal or Private?

Federal or Private?

Private

Received ARRA Funding?

Received ARRA Funding?

No

History/Related Projects

History/Related Projects

Efficacy of N-acetyl cysteine in autism | 146553 | 2012 | 201473
Efficacy of N-acetyl cysteine in autism | 146555 | 2013 | 201473

 
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