What we know: Therapeutics

Iker Spozio


Medical interventions

What we know

  1. Improvement in autism-related behaviors during fever offers hope that the syndrome can be reversed rapidly.
  2. Genetic or pharmacological intervention can reverse signs of autism in mouse models of fragile X and Rett syndrome. Adult as well as developing animals have shown this reversal, offering hope for gene therapy and pharmacological intervention.
  3. Oxytocin inhalation (or intravenous infusion) has produced modest and inconsistent results in measures of trust and social interaction. Several oxytocin trials in children, adolescents and adults with autism are underway.
  4. Risperidone and other antipsychotics reduce repetitive behaviors, hyperactivity, aggression and self-injury in some individuals with autism.
  5. In a double-blind placebo-controlled trial, the GABA mimetic bumetanide reduced autism traits in children.
  6. Arbaclofen, a GABA mimetic, showed some improvement in social cognition in a subset of individuals.
  7. Melatonin reduces sleep latency and increases total sleep time in about 60 percent of children with autism with reported chronic sleep difficulties.
  8. Carnitine replacement therapy may improve communication and sleep efficiency.

Behavioral interventions

Behavioral interventions fall into two categories: those that provide a holistic developmental framework, such as the Early Start Denver Model, and those based on the principles of applied behavioral analysis, such as UCLA/Lovaas approach.

What we know

  1. The Early Start Denver Model offers significant improvement in verbal intelligence after one year of intensive (40 hours per week) intervention. At two years, the improvement in intelligence persists, and researchers noted other improvements in language skills, activities of daily living and motor skills. They saw no change in the severity of autism, according to the Autism Diagnostic Observation Schedule, or degree of socialization even after two years of intensive therapy.
  2. The UCLA Model of Early Intensive Behavioral Intervention offers significant gains in adaptive behaviors, as shown through four years of follow-up testing.


What we know

  1. Transcranial magnetic stimulation at low frequency over the dorsolateral prefrontal cortex may reduce repetitive behaviors in high-functioning individuals with autism.

What is next?

  1. Can efficient clinical trial networks be developed that will standardize methodologies, patient populations and informed consent?
  2. Which biomarkers are most useful to monitor changes in the core symptoms of autism?
  3. What is the potential of GABA mimetics, oxytocin mimetics and other candidates, as they arise, in children and adults with autism?
  4. Can agents that emerge from genetic screens, such as carnitine and branched-chain amino acids, prove beneficial?
  5. Does transcranial magnetic stimulation improve social cognition in autism? If so, do the effects last beyond the period of stimulation?
  6. Can useful outcomes in early, ‘proof-of-concept’ clinical trials be defined?
  7. Can induced pluripotent stem cells be used to assay candidate drug therapies? Can appropriate assays in flies, worms, fish and other genetic-model organisms be developed?
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