Treating certain patients who have autism spectrum disorder (ASD) with a growth hormone can improve social impairment.
Researchers at the Icahn School of Medicine at Mount Sinai in New York City enrolled nine children with autism and Phelan-McDermid syndrome (PMS), a disorder with symptoms similar to autism caused by the deletion or mutation of a certain gene (SHANK3) on a particular chromosome (22).
The SHANK3 gene has an important role in the way synapses, the gaps between nerve cells involved in brain messaging, function, and has been examined for its role in autism.
The children were given three months of treatment with insulin-like growth factor-1 (IGF-1), and three months of placebo, in a random order. IGF-1 promotes nerve cell survival, synaptic maturation, and synaptic plasticity, the ability of synapses to strengthen or weaken over time, the researchers said.
When children were given IGF-1, they saw improvement in social behavior and restrictive behaviors, the researchers reported in the journal Molecular Autism.
“This clinical trial is part of a paradigm shift to develop targeted, disease modifying medicines specifically to treat the core symptoms of ASD,” Joseph Buxbaum, PhD, director of the Seaver Autism Center at Mount Sinai, said in a statement. “Results from this pilot trial will facilitate larger studies that more definitively inform efficacy and better targeted therapeutic treatments.”
New research has found that a growth hormone can significantly improve the social impairment associated with autism spectrum disorder in patients with a related genetic syndrome.
The pilot study, conducted at the Icahn School of Medicine at Mount Sinai, focused on the use of insulin-like growth factor-1 (IGF-1) to treat Phelan-McDermid syndrome (PMS), a disorder caused by a deletion or mutation of the SHANK3 gene on chromosome 22.
Along with facing developmental and language delays and motor skill deficits, most people with PMS also have autism spectrum disorder, according to the researchers.