First drug to demonstrate therapeutic effect in a type of autism identified

Washington, May 20 (ANI): American researchers have identified a drug that improves communication between nerve cells in a mouse model of Phelan-McDermid Syndrome (PMS).

Previous research has shown that a gene mutation in the brain called SHANK3 can cause absent or severely delayed language abilities, intellectual disability, and autism.

Researchers at Mount Sinai School of Medicine developed mice with a mutant SHANK3 gene and observed a lapse in communication between nerve cells in the brain, which can lead to learning problems. This communication breakdown indicated that the nerve cells were not maturing properly.

The scientists then injected the mice with a derivative of a compound called insulin-like growth factor-1 (IGF1), which is FDA-approved to treat growth failure in children.

After two weeks of treatment, nerve cell communication was normal and adaptation of nerve cells to stimulation, a key part of learning and memory, was restored.

Joseph Buxbaum, Director of the Seaver Autism Center for Research and Treatment at Mount Sinai School of Medicine, said: "The result of IGF1 treatment of these mice is an exciting development on the road to ultimate therapies for individuals with PMS.

"If these data are further verified in additional preclinical studies, individuals with a SHANK3 mutation may benefit from treatments with compounds like this one." (ANI)