Promising autism drug progresses to clinical study

A new clinical study will begin this month to evaluate the first drug shown to improve the symptoms of a subtype of autism. Researchers at Mount Sinai School of Medicine will look at the effect of insulin-like growth factor (IGF-1) in children who have SHANK3 deficiency (also called 22q13 Deletion Syndrome or Phelan-McDermid Syndrome), a known cause of autism spectrum disorder. The new study builds on previous research from 2010 which showed that after two weeks of IGF-1 treatment in a mouse model, deficits in nerve cell communication were reversed and deficiencies in adaptation of nerve cells to stimulation, a key part of learning and memory, were restored. IGF-1 is a US Food and Drug Administration-approved, commercially available compound that is known to promote neuronal cell survival as well as synaptic maturation and plasticity.