Autism Clinical Trial
Children with autism are often treated with psychiatric drugs. These medications have been
shown to improve their language and social function, and are important in improving their
quality of life. In many cases it is difficult to determine the best drug dose, and a
favorable response occurs in only 30%-70% of individuals, with many children suffering
significant adverse drug reactions.
Pharmacogenetics is the study of the role of different genes on drug behavior. The
cytochrome P450 is the most important enzyme, involved in the metabolism of a vast number of
drugs, including psychiatric medications. The multiple variations in this gene can result in
the different response observed in different patients, even when treated with similar doses
of the drug.
Hypothesis(es):
Mapping the different types of cytochrome P450 gene, in children with autistic disorders
will improve the rate of success of medical treatment, and prevent adverse drug reactions.
Potential Impact:
If successful, our study can help thousands of children and their families by developing a
system of "tailored medicine" that is based on the specific activity of the various enzymes
present in that particular patient. Better medical treatment will facilitate better daily
interactions with the children and enhance their quality of life. Furthermore, recognizing
children that are resistant to medication will prevent unnecessary use of drugs.
It should be noted this is the first study focusing on children receiving psychiatric
medications using pharmacogenetics. Found to be effective, this method can also be applied
to other groups of medications and to other patients.
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Observational Model: Cohort, Time Perspective: Prospective
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