Trial of Methyl B12 on Behavioral and Metabolic Measures in Children With Autism

Autism is a complex neurodevelopmental disorder with early childhood onset characterized by
impairments in communication, social interaction, and repetitive behavior. Due to the lack
of known treatments for autism, many parents seek complementary and alternative medical
(CAM) therapies hoping to help their affected child. Methylcobalamin (methyl B12) is a
commonly used CAM treatment that has anecdotal reports of remarkable clinical improvements
with few side effects. Prior studies have found that children with autism have deficiencies
in key metabolites and antioxidants which can be caused by methyl B12 deficiency; additional
studies have shown that methyl B12 normalizes deficiencies in these metabolites and
antioxidants. Based on these reports, a pilot study was conducted at UC Davis on the effect
of methyl B12 on the behavioral and metabolic measures in children with autism. The
preliminary results of 29 subjects revealed a subgroup of 9 responders to clinical behavior
assessments. These responders also demonstrated significant improvement on the plasma
measures of antioxidant capacity, suggesting methyl B12 improves symptoms in a subgroup of
children with autism by increasing key antioxidants. The current study will have an 8 week
double blind design with 50 subjects, designed to evaluate improvements from methyl B12 by
using behavioral assessments and analysis of specific metabolites in the subjects' blood.
This study will determine whether methyl B12 will lead to benefits in any of the core
features of autism, and will examine metabolic changes with the hope of potentially
identifying a biomarker for treatment response in a subgroup of subjects.

Inclusion Criteria:

- Diagnosis of DSM IV defined autism and meets cut off on Autism Diagnostic
Inventory-Revised (ADI-R) and/or the Autism Diagnostic Observation Scale (ADOS)

- Age 3 through 7 years

- IQ of 50 or above

- Parental agreement to continue present dietary, behavioral or psychotropic drug
treatment but not change treatment during 8 week intervention

- Willingness to have blood drawn, without the use of a sedative prescription from the
study doctor

Exclusion Criteria:

- Bleeding disorder

- Cancer

- Seizure disorder

- Fragile X or other known genetic cause of autism

- Perinatal brain injury (i.e.: cerebral palsy)

- Other serious medical illnesses

- Current use of any B12 supplement