Is there a basis for novel pharmacotherapy of autism?

Life Sci. 2000 Aug 25;67(14):1667-82. doi: 10.1016/s0024-3205(00)00763-3.


No medication has yet been shown to consistently alter the symptoms or the course of autism in the majority of patients. The present pharmacotherapy is mainly palliative and sometimes effective in attenuating specific behaviors. The search for better treatment involves examination of the underlying pathophysiology, the genetic or environmental etiology (including possible iatrogenic causes), and assessment of the clinically-generated evidence of efficacy, including serendipitous or unexplained findings. Subtle neuroanatomic and neurochemical changes are being explored and there are anecdotal reports or limited clinical trials that suggest some therapy might be possible. Secretin is a surprising recent addition to the list of candidates. The pharmacologic mechanism by which these agents might provide such effect is not clear, but hypotheses are beginning to emerge. In addition, the prevention of some uncertain number of autism cases is being investigated by examination of certain vaccinations as putative causative or contributory factors. These topics are reviewed in this article, which has the additional purpose of stimulating novel drug discovery efforts for this enigmatic disorder.

Publication types

  • Review

MeSH terms

  • Amino Acid Sequence
  • Animals
  • Antipsychotic Agents / therapeutic use
  • Autistic Disorder / drug therapy*
  • Autistic Disorder / etiology
  • Buspirone / therapeutic use
  • Dietary Supplements
  • Humans
  • Immunoglobulins, Intravenous / therapeutic use
  • Molecular Sequence Data
  • Naltrexone / therapeutic use
  • Narcotic Antagonists / therapeutic use
  • Risperidone / therapeutic use
  • Secretin / therapeutic use
  • Serotonin Receptor Agonists / therapeutic use
  • Serotonin Uptake Inhibitors / therapeutic use


  • Antipsychotic Agents
  • Immunoglobulins, Intravenous
  • Narcotic Antagonists
  • Serotonin Receptor Agonists
  • Serotonin Uptake Inhibitors
  • Secretin
  • Naltrexone
  • Risperidone
  • Buspirone